Stem Cell Clinic

Is Sight Care Legit? Review How SightCare Ingredients Work Before … – Bellevue Reporter

It is common knowledge today that your eye health suffers indiscriminately because of increased exposure to blue light emitted from electronic gadgets. Every day we are on our mobile phones or laptops, scrolling through either work or social media. This makes our eyesight more vulnerable and affects night vision and cognitive function.

You can rely on a supplement that claims to reverse poor eye health. Our team has identified the Sight Care eye health formula as one of the best ways to attain clear vision.

Sight Care can prove to be your go-to supplement if you spend your days in front of devices that emit blue light. The Sight Care formula comprises natural ingredients including minerals, herbs, roots, and plants. These ingredients are backed by science to produce impressive results and carry multiple health benefits for the human body.

It is a powerful supplement that helps you attain optimal eye health naturally. The formula of Sight Care has been developed from the work of a Nobel Prize winner who found a way to reverse age-related macular degeneration. This scientific vision breakthrough does not just improve visual acuity, it takes care of your overall health.

In this Sight Care review, we will explore all the details, big and small, about this supplement to help you make an informed choice about its purchase. But first, let us go through its summary below.

Category:

Dietary Supplement

Product Form:

Capsule

Serving Quantity:

60 capsules

Dosage Guideline:

Consume 2 capsules daily

Key Ingredients:

Astaxanthin, Quercetin, N-Acetyl-L-Cysteine, Zeaxanthin, Lutein, L-Lysine, Eyebright, Bilberry Fruit Extract, etc.

Product Characteristics:

Price:

$69 for one bottle (Official Website)

Key Benefits:

Bonus Products:

Yes

Money-Back Guarantee:

180-money-back guarantee

Sight Care is one of the best natural eye health supplements that is resplendent with features to benefit users immensely. This supplement can help you enjoy healthy vision without affecting other functions of your body. With the help of Sight Care capsules, you can notice significant improvements in your overall eye health. Lets go through the highlights of Sight Care below.

Made only using natural ingredients, Sight Care is a wonder for those people who easily catch side effects from artificial elements.

Sight Care is a peoples favorite because we found so many positive reviews about this supplement online.

The Sight Care eye health supplement works mainly to provide you with 20/20 vision and top-notch visual acuity so that you can differentiate between objects from far away.

If your night vision is suffering, you can use the exclusively designed formula of Sight Care which is scientifically backed to support optimal vision.

According to the Sight Care official website, this supplement is manufactured in FDA-registered and GMP-certified facilities in the USA.

The ingredients added in Sight Care are tested by third-party labs to protect you against side effects.

Get started today and see the difference SightCare can make >>>

The makers of Sight Care made a very important revelation through their research about the eye health of humans. According to them, the main reason behind poor eyesight is the low levels of adult repair stem cells after a certain age. Every person has these cells in abundance at a young age.

When your eyes suffer damage in your 20s or 30s, these cells transform themselves into healthy eye cells to keep your vision immaculate. According to the work of a Nobel Prize winner, these cells do not have any assigned role and make up for any damaged cells in the body.

So, as you grow older, you already use up most of these cells and as a result, develop age-related macular degeneration and poor night vision.

The Sight Care formula has been specially designed to boost the levels of adult repair stem cells using natural ingredients like astaxanthin, eyebright, quercetin, lutein, and zeaxanthin. It is one of the only natural eye health supplements to take this approach to improve your vision. As a result, Sight Care is popular among people who have increased exposure to blue light.

Sight Care ingredients work together to influence the growth of these cells so that you can experience healthy eyesight. These ingredients tell your body to produce brand-new stem cells to improve visual acuity and support clear vision. Several studies have proven the efficacy of Sight Care ingredients in improving stem cell potency.

This helps protect eye health and prevent age-related macular disease in old age. Sight Care is filled with nutrients that fight free radicals which in turn helps support eye health. This supplement improves blood circulation to the eye cells which allows the restoration of good vision at a cellular level. SightCare can protect you from blue light that is emitted from electronic devices.

The formula of SightCare is rich in antioxidants that support your cognitive function. By boosting blood flow in the body, Sight Care detoxifies your body and supports high energy levels. It can also help you adhere to a healthy lifestyle which helps promote your liver health. All in all, SightCare can prove highly beneficial for your overall well-being.

The formula of SightCare is rich in antioxidants that support your cognitive function. The connection between the eye and brain health is astonishing. When your brain receives proper nourishment, it prepares a foundation for better ocular health. Sight Care provides holistic support for superior brain health which paves the path for clear vision and better eye well-being.

Sight Care uses natural ingredients rich in amino acids which act as the building blocks for your brain cells. This tones down vision problems and helps improve visual acuity. Once your brain health becomes optimal, the eye tissues become stronger to support better vision.

One of the very few natural eye health supplements to support brain health and fight age-related eye complications, Sight Care improves your decision-making skills.

Sight Care Is On Sale Now For A Limited Time!

Sight Care supplements are brimming with ingredients that are very nourishing for your body. These capsules carry multiple health benefits for the human body. They can improve the well-being of your eyes, brain, liver, and immune system. Lets take a detailed look at the benefits of Sight Care.

This supplement supports healthy eyesight by boosting blood circulation in the body. It can help you in maintaining eye health so that you dont develop poor eyesight. This eye health formula can nourish your eye cells with better blood flow to offer improved eye health. It helps greatly in maintaining healthy vision and tackling vision problems.

Apart from promoting eye health, Sight Care also helps improve your brain health with the help of a nutrient-rich formulation. If you have poor eye health, it might be because of slow cognitive function and improper blood flow to the brain. Sight Care capsules help you attain optimal brain health to lead a healthy lifestyle.

The SightCare eye health supplement helps you maintain healthy eyesight by improving your visual acuity and supporting 20/20 vision. These capsules improve night vision and promote clear vision at all times to reduce the chances of vision impairment.

Sight Care supports a healthy inflammatory response to support optimal vision. It contains anti-inflammatory agents that support healthy vision in users.

This vision health supplement is well-formulated to improve your night vision and enhance visual acuity. SightCare helps protect eye health at nighttime using natural ingredients that boost healthy blood flow. It can help in maintaining night vision so that you can go out without anyones help.

The formula of the Sight Care vision supplement is rich in nutrients that are capable of boosting your energy levels. This vision health supplement can do more than just promote healthy vision so that you enjoy your life and lead a healthy lifestyle.

When you consume Sight Care supplements along with a balanced diet, it detoxifies your body and improves your liver health.

The natural ingredients present in the formula of this vision health supplement can nourish your body deeply. This supplement can improve your immunity and support brain function to support better overall health. You can notice a vast improvement in your general well-being after taking this dietary supplement daily.

To enjoy the benefits of SightCare, click here to order your supply now!

The natural ingredients in SightCare are backed by science. The ingredients, which include lutein and zeaxanthin, have been studied for their potential to improve overall eye health and help protect the eyes from certain conditions such as age-related macular degeneration (AMD). Below is an overview of some of these ingredients:

Astaxanthin consists of a long chain of conjugated double bonds with various functional groups attached, resulting in a highly complex structure. This compound is primarily found in certain types of microalgae, as well as in the marine animals that consume these algae.

It is able to neutralize harmful free radicals that are generated by exposure to ultraviolet (UV) radiation, environmental pollutants, and other sources of oxidative stress. By doing so, astaxanthin helps to protect the delicate tissues of the eye, including the retina, from damage caused by these free radicals, which can contribute to the development of age-related eye conditions such as macular degeneration and cataracts.

Furthermore, astaxanthin has been found to enhance blood flow to the retina and improve microcirculation within the eye. This can have a positive impact on visual acuity by ensuring an adequate supply of oxygen and nutrients to the cells of the retina, which are responsible for capturing light and transmitting visual signals to the brain.

N-Acetyl-L-Cysteine (NAC) is a powerful antioxidant that is added to Sight Care pills to promote eye health and boost night vision. This compound works by supporting the production of glutathione, a potent antioxidant that helps protect the eyes from oxidative stress and damage.

A clinical trial conducted by researchers at the University of Melbourne examined the effects of NAC supplementation on healthy individuals with impaired night vision. The study involved 52 participants who were randomly assigned to receive either NAC or a placebo for a period of three months. The participants night vision was assessed using a device that measured their ability to detect low levels of light.

The results of the study showed that the group receiving NAC supplementation experienced a significant improvement in night vision compared to the placebo group. Specifically, the participants who took NAC had a 45% improvement in their ability to detect low levels of light, while the placebo group showed no significant improvement.

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Eyebright, also known by its botanical name Euphrasia officinalis, is a small flowering plant that belongs to the Orobanchaceae family. It is native to Europe and has been used for centuries as a herbal remedy for various eye conditions. Eyebright gets its name from the belief that its flowers resemble an eye, with a dark spot at the center resembling the pupil.

From a scientific perspective, Eyebright contains several bioactive compounds, such as flavonoids, iridoid glycosides, and tannins, which contribute to its beneficial effects on mental sharpness and eye health.

Moreover, iridoid glycosides found in Eyebright possess anti-inflammatory properties that can benefit both mental sharpness and eye health.

By inhibiting inflammatory pathways, iridoid glycosides in Eyebright help to reduce inflammation in the brain, thereby supporting mental clarity and cognitive performance.

Bilberry fruit, known for its vibrant blue color and small size, resembles a blueberry but with a slightly darker hue. This small, power-packed berry has been used for centuries to alleviate signs of eye cell inflammation and boost overall vision.

A study involved 36 participants with early signs of age-related macular degeneration (AMD), a condition characterized by the deterioration of the central part of the retina. The participants were divided into two groups, with one group receiving bilberry extract and the other group receiving a placebo.

The study reported a 63% improvement in visual acuity and a 42% increase in macular pigment density in the bilberry group, compared to the placebo group.

Lutein and zeaxanthin act as natural antioxidants, which means they have the ability to neutralize harmful free radicals in the eye. A clinical trial conducted by the Age-Related Eye Disease Study 2 (AREDS2) Research Group demonstrated the effectiveness of these carotenoids in reducing the risk of advanced age-related macular degeneration (AMD).

The study showed that a daily supplement containing lutein (10 mg) and zeaxanthin (2 mg) significantly reduced the progression to advanced AMD by 18% in high-risk individuals.

A study published in the Journal of Optometry investigated the effects of lutein and zeaxanthin supplementation on visual function in healthy young individuals. The participants who received a supplement containing lutein (10 mg) and zeaxanthin (2 mg) for six months experienced a significant improvement in contrast sensitivity and glare recovery compared to the placebo group.

Contrast sensitivity refers to the ability to distinguish objects of similar brightness, while glare recovery is the ability to regain clear vision after exposure to bright lights.

Dont miss out on the benefits that Sight Care can provide order now!

SightCare is an amazing eye health supplement that can improve your visual acuity and prevent the onset of age-related eye diseases in older adults. There are many Sight Care reviews online where people have left admiring comments about this supplement. You will find this Sight Care review particularly insightful.

I was looking for a supplement to combat vision impairment and I came across SightCare. After taking it for a short time, I noticed a stark difference in my ocular health and I was impressed beyond words. As time went by, my eyesight improved and I have a perfect 20/20 vision now.

Sight Care is a pretty safe eye health supplement because it does not contain GMOs, gluten, dairy, stimulants, or chemical compounds in its formulation. The usage of 100% natural ingredients in Sight Care makes it safe for everyday intake for enhanced visual clarity.

Moreover, SightCare is manufactured in GMP-certified and FDA-registered facilities in the USA and tested for purity and quality. There are also no cases of side effects according to multiple positive Sight Care reviews.

There are 60 capsules in every bottle of the Sight Care supplement. When we looked through the official website of this supplement, we found that you need to take two petite Sight Care capsules every day to deal with vision problems and maintain healthy eyesight.

The Sight Care supplement makes use of blue light-obstructing ingredients that work efficiently to help maintain optimal eye health in users. This supplement can prevent age-related vision problems by blocking damage from blue light emitted from electronic devices. This blue light can cause age-related macular degeneration and degrade your eyesight.

According to the official website of Sight Care, this supplement helps in maintaining optimal eye health by reversing the damage caused by blue light.

You can find SightCare on its official website. Every bottle of this vision health supplement contains 30 servings and is available for $69. You can also buy this supplement for a lower price if you buy it in bulk.

If you want crystal clear vision, you must order the 3-bottle pack of SightCare for $177 ($59/per bottle). But, if you want improved overall eye health in the long run, you must order the 6-bottle pack of Sight Care for $294 ($49/per bottle).

The makers of Sight Care offer a whopping 180-day money-back guarantee on every order of this supplement from its official website. Every person is eligible for this 180-day money-back guarantee with no exceptions.

If you dont get any relief from age-related eye diseases, you can avail of this 180-day money-back guarantee to claim your refund. We should warn you that this 180-day money-back guarantee is only valid on the official website of Sight Care.

The Sight Care vision supplement comes with a bonus book on every three-bottle and six-bottle purchase from its official website. This bonus book is The Truth About Vision and it helps you to promote vision health naturally. You also get access to a private VIP Client Area where you can gain resources, guides, and videos about improving eye health.

Sight Care is one of the many natural eye health supplements available on the market. There are other supplements or eye vitamins that claim to enhance vision and tackle age-related vision diseases. We compared SightCare with these supplements to identify its strengths and weaknesses.

Divine Vision 12 is a well-known vision supplement that promises to promote optimal eye health in daily users. This supplement contains natural ingredients like bilberry and grape seed which are also found in the formula of Sight Care. Together with other ingredients, they improve vision and support optimal brain health.

This supplement blocks out the damaging blue light from digital devices to protect you against age-related macular disease. However, the benefits of Divine Vision 12 do not encapsulate your overall health. Unlike it, SightCare boosts your brain health, liver health, and immune health. Furthermore, it works to enhance adult repair stem cells which is scientifically proven to improve vision.

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Eyesight Max is another vision health supplement that claims to support healthy eye function in users. The working of Eyesight Max is different from Sight Care. According to the makers of Eyesight Max, the main reason behind vision loss is PM 2.5 particles which are dangerous pollutants found everywhere in the atmosphere.

These particles damage your brain health which in turn worsens your vision. When these particles enter your blood circulation, your eyes suffer extensive damage. To support healthy vision, Eyesight Max forms a natural immune barrier. It helps cleanse your body of toxins to reduce the risk of developing age-related macular degeneration.

Both natural eye health supplements, Eyesight Max and SightCare enhance visual acuity and promote your overall well-being. However, there are no bonus books available with Eyesight Max. But, you get The Truth About Vision with Sight Care to improve your eyesight.

The last eye health supplement that we will compare Sight Care with is ReVision. It is a natural supplement that contains vitamins and minerals to boost blood flow to your eyes and improve your ocular health. Although it contains some of the same ingredients as SightCare, there is nothing special about the working of the ReVision formula.

Sight Care, on the other hand, aims to boost the levels of adult repair stem cells which are the newly discovered cause of vision loss in older adults. Low levels of these cells can lead to age-related macular disease and worsen your visual clarity. The formula of SightCare helps in promoting your vision well-being to block out the damage caused by blue light.

This supplement also provides other health benefits like improved energy levels and liver health. It also helps in promoting healthy brain function and improving eye-brain coordination.

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Ensuring optimal vision goes beyond routine eye check-ups; its intrinsically linked to what we fuel our bodies with. A balanced diet rich in essential nutrients significantly contributes to maintaining healthy eyes.

Vitamin A takes the spotlight, playing a pivotal role in preventing night blindness and promoting overall eye health. Leafy greens, carrots, and sweet potatoes are rich sources. Equally crucial are antioxidants like lutein and zeaxanthin found in kale, spinach, and eggs, safeguarding against age-related macular degeneration.

Omega-3 fatty acids, found in fish like salmon and flaxseeds, nurture the eyes cell membranes. Zinc, prevalent in meat and dairy, supports the health of the retina. Antioxidant-rich fruits like berries add another layer of defense.

In essence, a diet encompassing a spectrum of colorful fruits, vegetables, and nutrient-dense proteins ensures a holistic approach to eye health, fortifying your vision for the long haul.

Recognizing signs of deteriorating eye health is paramount for timely intervention. Regular self-assessment can be a powerful tool. If you notice persistent blurriness, difficulty seeing in low light, or sudden changes in vision, its a clarion call to schedule an eye examination.

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Is Sight Care Legit? Review How SightCare Ingredients Work Before ... - Bellevue Reporter

T-Cell Malignancies Reported in Patients Who Received BCMA … – OncLive

The FDA has received reports of T-cell malignancies in patients who have been treated with CD19- or BCMA-directed autologous CAR T-cell immunotherapies.1 These T-cell malignancies include CAR-positive lymphoma and were reported from postmarketing adverse effect (AE) data sources and clinical trials in patients who received several CAR T-cell products in the class.

The FDA has determined that the risk of T-cell malignancies is apparent in all currently approved CD19- and BCMA-directed genetically modified autologous CAR T-cell immunotherapies, including:

The FDA emphasizes that although the benefits of these products in their indicated uses continue to outweigh their potential risks, it is investigating the identified risk of T-cell malignancy, which may have serious outcomes, such as hospitalization and death.1 The agency is evaluating whether regulatory action pertaining to this risk is necessary.

All gene therapy products with integrating lentiviral or retroviral vectors, including CD19- and BCMA-directed genetically modified autologous T-cell immunotherapies, are labeled with a United States prescribing information class warning for the development of secondary malignancies. The initial FDA approvals of these products included postmarketing requirements per Section 505(o) of the Federal Food, Drug, and Cosmetic Act for investigators to conduct 15-year observational follow-up safety studies for the purpose of assessing the long-term safety profiles of these products and the risk of developing secondary malignancies after treatment.

Patients being treated with these CD19- and BCMA-directed genetically modified autologous T-cell immunotherapies should receive life-long monitoring for the development of new malignancies. If a patient develops a new malignancy after receiving treatment with these products, they should contact the respective products manufacturer to report the incident and receive instructions regarding the collection of patient samples that will be tested for the presence of the CAR transgene.

Suspected AEs, including T-cell malignancies, related to these products can be reported to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.

Health care providers, clinical investigators, caregivers, and patients with questions about these products can contact the FDAs Center for Biologics Evaluation and Research atocod@fda.hhs.gov.

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T-Cell Malignancies Reported in Patients Who Received BCMA ... - OncLive

Comparison of fludarabine/melphalan (FluMel) with fludarabine … – Nature.com

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Comparison of fludarabine/melphalan (FluMel) with fludarabine ... - Nature.com

NOT-AR-23-022: Request for Information on Themes for the NIAMS … – National Institutes of Health (.gov)

Request for Information on Themes for the NIAMS Strategic Plan for Fiscal Years 2025-2029

The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) supports research into the causes, treatment, and prevention of arthritis and musculoskeletal and skin diseases; the training of basic and clinical scientists to carry out this research; and the dissemination of information on research progress in these diseases. NIAMS is updating its Strategic Plan to help guide the research, training, and information dissemination programs it supports between fiscal years 2025 through 2029. The new Plan will focus on cross-cutting thematic research opportunities that position the Institute to make a difference in the lives of all Americans.Because public input is a crucial step in this effort, the Institute issued a Request for Information (NOT-AR-22-023) and hosted a meeting attended by approximately 160 researchers, patient representatives, and staff from other Federal entities to gain insight into topics that could be included in the new Strategic Plan.

Through this Request for Information, NIAMS invites feedback from researchers in academia and industry, health care professionals, patient advocates and health advocacy organizations, scientific or professional organizations, Federal agencies, and other interested members of the public on the Institutes distillation of the input received to date. Professional societies and patient organizations are strongly encouraged to submit a single response that reflects the views of their entire membership.

Please provide your perspective on the following potential cross-cutting themes, examples, and bold aspirations. NIAMS is particularly interested in suggestions for additional or alternative:

Examples:

Bold Aspirations:

Examples:

Bold Aspiration:

Note: Efforts to identify and reduce health disparities and provide all Americans with equitable access to clinical and epidemiologic studies and healthcare should be considered for NIAMS-funded research projects whenever possible.

Examples:

Bold Aspirations:

Examples:

Bold Aspiration:

Note: Consistent with the note under Health disparities and health equity, studies of lifestyle factors and environmental exposures should include efforts to identify and reduce health disparities and provide all Americans with equitable access to clinical and epidemiologic studies and healthcare whenever possible.

Examples:

Bold Aspiration:

Note: Consistent with the note under Health disparities and health equity, clinical and epidemiologic research should include efforts to identify and reduce health disparities and provide all Americans with equitable access to clinical and epidemiologic studies and healthcare whenever possible.

Examples:

Bold Aspiration:

Examples:

Bold Aspiration:

Note: Training and workforce efforts are essential for the pursuit of all cross-cutting thematic research areas in the new NIAMS Strategic Plan.

Examples:

Bold Aspiration:

Examples:

Bold Aspirations:

Responses to this RFI must be submitted electronically at https://rfi.grants.nih.gov/?s=654a7bc81e7ccb6f7d03d792.

Responses must be received by Monday, January 1, 2024.

Responses to this RFI are voluntary. Do not include any proprietary, classified, confidential, trade secret, or sensitive information in your response. The responses will be reviewed by NIAMS staff, leadership, and Advisory Council members. Individual feedback will not be provided to any respondent. NIAMS will use the information submitted in response to this RFI at its discretion and will not provide comments to any respondents submission. Respondents are advised that the Government is under no obligation to acknowledge receipt of the information received or provide feedback to respondents with respect to any information submitted.The Government reserves the right to use any submitted information on public NIH websites, in reports, in summaries of the state of the science, in any possible resultant solicitation(s), grant(s), or cooperative agreement(s), or in the development of future funding opportunity announcements.

This RFI is for information and planning purposes only and shall not be construed as a solicitation, grant, or cooperative agreement, or as an obligation on the part of the Federal Government, the NIH, or individual NIH Institutes and Centers to provide support for any ideas identified in response to it. The Government will not pay for the preparation of any information submitted or for the Governments use of such information. No basis for claims against the U.S. Government shall arise as a result of a response to this request for information or from the Governments use of such information.

We look forward to your input and hope that you will share this RFI document with your colleagues.

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NOT-AR-23-022: Request for Information on Themes for the NIAMS ... - National Institutes of Health (.gov)

Dr Hurwitz on Ongoing Investigations of the Use of CAR T-Cell … – OncLive

Michael Hurwitz, MD, PhD, associate professor, internal medicine (medical oncology), Yale School of Medicine, discusses the ongoing investigation into the use of CAR T-cell therapies in patients with solid tumors, such as kidney cancers.

Hurwitz begins by stating that considerations surrounding the use of CAR T-cell therapy in solid tumors, such as renal cell carcinoma (RCC), have been uncertain. The phase 1 COBALT-RCC trial (NCT04438083), which investigated CTX130 allogeneic CRISPR/Cas9engineered CAR T-cell therapy in patients with advanced clear cell RCC, is currently inactive. However, a new agent with similar attributes to the CAR T-cell product investigated in COBALT-RCC is under development and may improve upon the outcomes seen in COBALT-RCC, Hurwitz begins.

Another trial, the phase 1 TRAVERSE trial (NCT04696731), is ongoing at some sites, he explains. This trial involves off-the-shelf CAR T-cell therapy, Hurwitz explains. These modified CAR T cells are engineered to evade the recipient's immune response and eliminate the need for personalized CAR T-cell production, offering a faster turnaround that is crucial for individuals with advanced solid tumors, Hurwitz explains.Traditionally, introducing foreign T cells into the body triggers immune responses, which are addressed by removing human leukocyte antigens, so the body does not recognize the T cells as foreign. In these modified CAR T cells, the endogenous T-cell receptors are also removed, ensuring these cells do not perceive the body as foreign, he expands.

Along with the FDA approvals of CAR T cells for patients with hematologic malignancies, their application in solid tumors is evolving, Hurwitz emphasizes. Ongoing preclinical research aims to engineer safe, specific, and effective CAR T cells, he states.

These innovations with CAR T-cell therapy promise highly targeted, safe cancer treatments for patients with solid tumors, Hurwitz continues. Looking forward, the possibility of synergies between CAR T-cell therapy and other treatments looms, he notes. Although the timing of integrating CAR T-cell therapies into the solid tumor treatment armamentarium is uncertain, combining these products with other agents offers a glimpse into a future where cancer treatment is more effective and personalized.In essence, technological advances in cancer therapy are just beginning to unfold, Hurwitz adds. The future promises innovations and a convergence of technologies to reshape cancer treatment, ushering in an era of hope and healing, he concludes.

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Fish-like genetic program used to turn human retinal cells into neurons – EurekAlert

image:

Overall, our study provides a proof-of-principle that human glia can be reprogrammed to cells that are capable of making new neurons," said Thomas Reh, PhD, University of Washington, USA. "This opens up an entirely new way to repair the retina in people that have lost neurons to disease or trauma."

Credit: Thomas Reh, PhD

Loss of neurons in in the retina due to trauma or disease leads to vision impairment or blindness, a process which is irreversible in humans. Interestingly, some animals like fish have the built-in ability to regenerate retinal neurons by turning another retinal cell type called Muller glia into neurons. This conversion does not happen spontaneously in humans and other mammals, but new research by Thomas Reh, Juliette Wohlschlegel, and colleagues at the University of Washington, USA, published in the journalStem Cell Reports, shows that human Muller glia can be coaxed into changing identity in the laboratory, which could serve as a potential source of new neurons to treat vision loss.

"Overall, our study provides a proof-of-principle that human glia can be reprogrammed to cells that are capable of making new neurons,said Thomas Reh, PhD, University of Washington."This opens up an entirely new way to repair the retina in people that have lost neurons to disease or trauma."

Muller glia are supportive cells in the retina which help photoreceptors and other retinal neurons to function properly. In some species like fish and birds, Muller glia turn into immature retinal cells upon injury and subsequently generate new retinal neurons. By contrast, Muller glia in the mammalian retina react to injury with scar formation and inflammation without making new neurons. This difference in behavior is based on different genetic programs being activated in fish versus mammalian Muller glia after injury. Artificial activation of a fish-like genetic program can turn mouse Muller glia into retinal neurons according to prior research. However, up until now, it has not been known if the same strategy can be used to convert human Muller glia into neurons. To answer this question, the researchers genetically modified human Muller glia in the lab to switch on neurons-specific genetic programs, as it naturally happens in fish. Indeed, within a week, the genetically modified cells adopted a neuron-like characteristics resembling immature retinal neurons. These findings suggest that human Muller glia can be coaxed into neurons and may serve as a resource to generate new neurons in patients retinas in the future. Of note, Muller glia in this study were derived from immature Muller glia and it remains to be seen if similar approaches can transform adult human Muller glia into neurons, and to what efficiency.

Stem Cell Reports

ASCL1 induces neurogenesis in human Muller glia

30-Nov-2023

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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Fish-like genetic program used to turn human retinal cells into neurons - EurekAlert

Sickle Cell Disease and Genetics: Understanding the Cause and… – Sickle Cell Disease News

This content is sponsored by bluebird bio, Inc. (bbb) and is intended for US patient/caregiver audiences 18 years of age and older only. Any other present or future content posted by a contributor, not expressly designated as bluebird bio, Inc.sponsored content, is not associated with bbb.

As a progressive, unpredictable disease, sickle cell is a genetic condition that affects everyone differently, which can make it difficult to understand. Simply learning more about how the body works can help explain the cause of sickle cell. And with a better understanding of how this disease works and how genetics are involved, you can make informed decisions with your doctor about how to navigate treatment.

As the name implies, sickle cell disease involves your cells. Each person has trillions of cells in their body, all working together to perform functions that keep us alive. Within each cell is DNA, the molecule that contains the genetic information for our cells, including genes. Genes provide the instructions for your cells. They help make the proteins that keep us healthy by powering muscles, attacking invading bacteria, or delivering oxygen throughout the body. Since each cell in your body relies on thousands of proteins to work properly in order for the cells to function correctly, your genes are the blueprint, or instruction manual, for your body. When theres a mutation (or change) in a gene, the instructions can cause a cell or protein to not function properly and potentially cause diseasesuch as sickle cell.

Check out the video to learn more about how genes and proteins impact the symptoms and complications of sickle cell.

There are many different types of cells, but with sickle cell disease its the red blood cells, the most common type of cell in your blood, that are affected. These cells are responsible for delivering oxygen throughout the body, which fuels your organs by giving them the energy they need to function. The protein within these cells that performs this function is called hemoglobin, which is made up of beta-globin proteins and alpha-globin proteins. In order for normal adult hemoglobin (HbA) to work properly, there must be a balance of functioning beta-globin and alpha-globin. The HBB gene provides the instructions for these proteins within hemoglobin, and a mutation in that specific gene affects the instructions, causing your body to produce an abnormal form of beta-globin, such as hemoglobin sickle (HbS), which results in a person having sickle cell disease or carrying the sickle cell trait.

Since sickle cell disease affects everyone differently, its important to develop a comprehensive care plan that works for you and your healthcare team. This plan should aim to monitor symptoms, address chronic complications, and manage your overall health. Its also important to build a healthcare team that includes specialists from different areas who can make your care plan work for you. This team should include your primary care doctor but can also include a hematologist (who specializes in diseases of the blood), a genetic counselor (who can help with family planning), and a community health worker (who may be able to assist you with education and day-to-day monitoring by working with local hospitals), among others.

Pain, fatigue, and other unpredictable symptoms of sickle cell can affect many different aspects of your life. The daily tasks of family life, work, and/or school, as well as future plans, can all be impacted by symptoms and complications, whether theyre sudden (acute) or ongoing (chronic). Knowing how to manage these symptoms and complications can help you or your loved ones better navigate sickle cell care.

Progress continues to be made in the treatment of sickle cell disease, but current options mainly help relieve symptoms and require lifelong use. These treatment options include oral medications and infusions, blood transfusions, and, for a limited number of patients, a hematopoietic stem cell transplant (which can lead to a cure; however, they are mostly limited to people who are under the age of 18 and have a matched-related donor available).

There are also developing therapies now being explored to address unmet needs in the management and treatment of sickle cell. One therapy approach being researched is gene therapy,* which is designed to treat sickle cell at the genetic level with the goal of changing the course of the disease.

Taking the time to educate yourself on the genetics of sickle cell is an important step in managing sickle celland remember, youre not alone. Beyond your loved ones and care team, there is an entire sickle cell community, including advocacy organizations, who are here to help. For more information, visit SparkSickleCellChange.com, a website developed by bluebird bio, to stay proactive in sickle cell care and planning for the future.

*Gene therapies for sickle cell are investigational and not FDA approved. Safety and efficacy have not been established.

bluebird bio is a trademark of bluebird bio, Inc.

2023 bluebird bio, Inc. All rights reserved. SCD-US-00280 08/23

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Sickle Cell Disease and Genetics: Understanding the Cause and... - Sickle Cell Disease News

FDA Approves Nirogacestat for Patients With Desmoid Tumors – Targeted Oncology

The FDA has granted approval to nirogacestat for the treatment of adult patients with desmoid tumors.1

Findings from the phase 3 DeFi trial support this approval as in the study, nirogacestat reduced the risk of disease progression or death by 71% compared with placebo among patients with desmoid tumors (HR, 0.29; 95% CI, 0.15-0.55; P < .001).2

This study is the first positive study for a gamma secretase inhibitor in any indication, and it's the largest and the most rigorous and robust clinical trial ever done in desmoid tumor, said Bernd Kasper, MD, PhD, professor, Mannheim University Medical Center, in an interview with Targeted Oncology. Nirogacestat demonstrated rapid, sustained and statistically significant improvements in all primary and secondary endpoints with a manageable safety profile. So it is definitely a candidate for register for registration in this indication. And it will probably be the standard of care for decimo to most in need of systemic therapy.

Among those given nirogacestat (n = 70), the Kaplan-Meier-estimated median PFS was not estimated and patients treated with placebo (n = 72) had a median PFS of 15.1 months (95% CI, 8.4-not estimable). The likelihood of being event-free at 1 year was higher among patients treated with nirogacestat vs placebo, at 85% (95% CI, 73%-92%) and 53% (95% CI, 40%-64%), respectively.

Additionally, event-free survival rates at 2 years were 76% (95% CI, 61%-87%) with nirogacestat and 44% (95% CI, 32%-56%) with placebo, respectively. The confirmed ORR was 41% with nirogacestat vs 8% with placebo (P < .001), and the complete response rates were 7% and 0%, respectively.

The median time to confirm first response was 5.6 months and 11.1 for the investigative vs control arm, respectively, and the median best percent change in target tumor size was -27.1% (range, -100 to 37) vs 2.3% (range, -100 to 47).

Any-grade adverse events (AEs) were observed in 100% of patients in the nirogacestat arm compared with 96% in the placebo arm, with grade 3 or greater AEs observed in 55% vs 17% of patients in the nirogacestat vs placebo arms, respectively.

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FDA Approves Nirogacestat for Patients With Desmoid Tumors - Targeted Oncology

Gene-editing therapy by Vertex and CRISPR poised for FDA approval – The Boston Globe

Its kind of surreal, said Tornyenu, 22, who grew up in Bethlehem, Pa., and participated in a clinical trial at Childrens Hospital of Philadelphia. Im, like, wait, I dont have sickle cell anymore.

The life-changing drug, developed by Boston-based Vertex and its Swiss partner CRISPR Therapeutics, is expected to be approved by the Food and Drug Administration by Friday for people with severe cases of the disease. Called Casgevy, it would usher in a new era not only for those with sickle cell but also for medicine: The drug would be the first gene-editing therapy authorized by US regulators, and uses a tool called CRISPR.

The likely approval Casgevy was cleared by British regulators last month raises both the promise of cures for diseases as well as the ethical concerns that come with the power to manipulate the building blocks of human life. With an expected price tag in the seven figures, it also touches on issues of equity in medicine.

Sickle cell primarily afflicts people of African descent. Research on the disease languished for decades, which many experts blame on structural racism, particularly in funding.

For Tornyenu, Casgevy has meant an end to the searing pain crises that caused her to miss a week of classes every month as a high school senior. After getting the treatment, she took the spring semester off from Cornell to recover from the debilitating effects of chemotherapy that made room in her blood marrow for gene-edited cells.

But now she no longer dreads the arrival of cold weather, which would often induce excruciating pain in her hips and legs. A senior at Cornell, she has a job lined up as a consultant at PricewaterhouseCoopers in Boston after graduation.

Im very hopeful [Casgevy] will be approved, she said, because I dont know what I would have done otherwise.

The gene-editing method that became known as CRISPR was first reported in a landmark 2012 paper by American biochemist Jennifer Doudna of the University of California, Berkeley, and French microbiologist Emmanuelle Charpentier of the Max Planck Institute for Infection Biology. They would share the 2020 Nobel Prize in chemistry for their work on the tool.

Sickle cell was an obvious choice for scientists to tackle with CRISPR. It was the first human disorder understood on a molecular level, its underpinnings explained in a landmark 1949 paper written by the future two-time Nobel laureate Linus Pauling. Yet progress against the disease was slow for decades afterward.

Sickle cell affects hemoglobin, the oxygen-carrying protein in red blood cells. It causes the round, flexible blood cells to deform into a sickle shape and stick to vessel walls. That deprives tissues of oxygen, causing crushing pain that can often only be relieved with opioids and blood transfusions.

Sickle cell can also lead to strokes, damage organs, and cause early death. A 2019 study in JAMA Network Open estimated the life expectancy of adults with sickle cell in the US is 54 years, about 20 years shorter than the general population.

In a clinical trial, Casgevy demonstrated remarkable results. The medicine completely relieved 29 of 30 sickle cell patients of debilitating episodes of pain for at least one year among trial participants who were followed for at least 18 months, according to Vertex. The patients received a one-time intravenous infusion of edited stem cells that flipped a genetic switch to restore their blood cells ability to carry oxygen throughout their bodies.

This is what a potential cure looks like, said Dr. Stephan Grupp, chief of the Cellular Therapy and Transplant Section at Childrens Hospital of Philadelphia. He was the principal investigator at the trial site where Tornyenu got Casgevy and was paid by Vertex to help organize the study at locations across the US.

About 100,000 Americans, most of them Black or Hispanic, are believed to have sickle cell. The Vertex-CRISPR treatment was geared for those with severe and repeated pain crises, roughly 20,000 people in the US. As of 2021, almost 8 million people around the world live with sickle cell, according to the Institute for Health Metrics and Evaluation at the University of Washington in Seattle.

The FDA has approved four medicines for the disorder, but none has been remotely as effective as Casgevy, which is expected to cost more than $1 million for a one-time infusion in the US, according to experts. (No price has been announced in the United Kingdom.) Sickle cell can be cured with a bone-marrow transplant, but few patients have compatible donors.

Patients are already inquiring about Casgevy, said Dr. Sharl Azar, a hematologist at Massachusetts General Hospital and medical director of its Comprehensive Sickle Cell Disease Treatment Center. He said he is eager to see whether the FDA clears it, how broad the approval would be, and whether Medicare and Medicaid would cover it.

Theres a lot of unknowns that were looking forward to working out in the coming months, he said. But I think everyone, from patients to providers, recognizes that this is a big deal.

Rahman Oladigbolu, a 52-year-old Harvard-educated filmmaker in Brockton, is among local patients interested in Casgevy. He has had six joints his hips, shoulders, and knees surgically replaced since 2000 because of damage from sickle cell. He walks with a cane at times, often gets lightheaded, and takes opioids to relieve persistent pain.

When Oladigbolu was growing up in Nigeria, his grandmother would take him to traditional medicine men and medicine women who prescribed herbs and potions, some of which they rubbed into his aching limbs after cutting him with a razor blade. He moved to the US when he was 28 and currently takes a sickle cell drug called crizanlizumab, which reduces his pain but doesnt eliminate it.

Pursuing a cure has been like a side job all my life, said Oladigbolu, who receives treatment at Boston Medical Center.

CRISPR-based treatments will likely be approved for other disorders in the coming years, experts say, although its hard to predict for what and when. Researchers, including scientists at multiple biotech companies and hospitals in Massachusetts, are studying the potential of gene editing for a variety of diseases, from ALS to forms of cancer.

There will be other gene-editing therapies, certainly, but each disease is different, said Dr. Stuart Orkin, a researcher at Dana-Farber Cancer Institute and Boston Childrens Hospital who in 2008 helped identify the gene that Casgevy snips to treat sickle cell. For some diseases, its not clear what to edit. People will argue about whats the right target. Each one is a special case.

Gene editing has also raised ethical concerns. In 2018, a Chinese scientist, He Jiankui, was widely condemned when he announced that he used CRISPR to edit DNA in human embryos to try to make them immune to HIV. The experiment sparked fears that He had opened the door to creating so-called designer babies children whose genetic makeup is altered to produce desired traits.

Dr. George Q. Daley, dean of Harvard Medical School, was among those who said Hes experiment raised the specter of a Brave New World of eugenics. Casgevy, he said recently, is completely different. The modifications it makes to DNA only helps sickle cell patients and cannot be passed on to their children.

Daleys bigger worry concerns access to Casgevy. While wealthy countries like the US have hospitals and doctors capable of preparing patients for the treatment and administering it, he said, millions of people with sickle cell in sub-Saharan Africa dont have those options.

This is a triumph of modern biomedicine, he said. The major ethical concerns now are issues of cost and equitable distribution.

Casgevy isnt the only gene-based medicine on the horizon for sickle cell. The Somerville biotech Bluebird Bio hopes the FDA approves a so-called gene therapy, lovo-cel, by Dec. 20. It also proved remarkably effective in clinical trials.

Unlike Casgevy, which cuts a gene, lovo-cel adds a modified gene into a patients DNA to enable blood cells to deliver oxygen. The FDA has approved at least eight gene therapies for mostly rare diseases since 2017.

Both Casgevy and lovo-cel are expected to be breathtakingly expensive. That has renewed questions about whether the health care system can afford such cutting-edge medicines.

Still, the Institute for Clinical and Economic Review, or ICER, an independent Boston-based drug-pricing watchdog, estimates that either drug could cost nearly $2 million and be worth it, given the cumulative costs of treating sickle cell over a lifetime and the benefits the new approaches would bring to patients and families.

Casgevy, which was called exa-cel in clinical trials, works by editing a patients bone marrow stem cells to make high levels of fetal hemoglobin the healthy, oxygen-carrying form of hemoglobin produced during fetal development that is replaced by adult hemoglobin soon after birth.

Unlike adult hemoglobin, fetal hemoglobin resists forming a crescent shape in sickle cell patients, and scientists have long searched for a way to restart it. The researchers behind Casgevy solved the problem by editing a gene called BCL11A, which regulates fetal hemoglobin.

The treatment involves multiple steps over several months. Patients must donate stem cells to be modified at a laboratory. Then donors have to undergo a grueling regimen of chemotherapy to make room in their bone marrow for the genetically altered cells. Finally, the patients get the cells back through a single infusion.

Dr. David Altshuler, Vertexs chief scientific officer, acknowledged that the gene-editing treatment is extremely complex and resource intensive. He said Vertex is researching the possibility of developing a pill that could do what Casgevy does without gene editing. (Vertexs business partner for Casgevy, CRISPR Therapeutics, is based in Zug, Switzerland, but has most of its workforce in Boston.)

FDA officials have raised concerns about the possibility that Casgevy could inadvertently change patients DNA beyond the targeted disease so-called off-target editing. Dr. Daniel E. Bauer, a staff physician at Dana-Farber Cancer Institute and Boston Childrens Hospital, told an FDA advisory panel on Oct. 31 that Casgevy contains hundreds of millions of edited cells and one could undoubtedly go off target and cause leukemia. But he described the risk as modest given the benefits of the treatment.

Altshuler said recently that there is no evidence to date of off-target editing, but it is important to be humble and to continue to follow patients. Vertex and CRISPR have pledged to follow trial participants for 15 years to make sure they stay healthy.

Tornyenu, the Cornell student, says she considers Casgevy a miracle and would celebrate Dec. 8 every year if the FDA approves the drug that day.

For lack of a better term, she said, its a big FU to sickle cell.

Jonathan Saltzman can be reached at jonathan.saltzman@globe.com.

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Gene-editing therapy by Vertex and CRISPR poised for FDA approval - The Boston Globe

Major Breakthrough: UofM Research Team 3D Prints Heart Valves – Mirage News

Credit: CHU Sainte-Justine

In a breakthrough in pediatric cardiac science, Canadian researchers have successfully produced a bio-ink that could someday be used to print functional, durable heart valves, offering hope for improving the prognosis of children with heart defects.

The discovery of a way to 3D print functional heart valves was made at the CHU Sainte-Justine Research Centre by Universit de Montral assistant medical professor Houman Savoji and his PhD student Arman Jafari.

The results of their research are published in the journal Advanced Functional Materials.

Tissue engineering can be used to create living tissues and organs by combining biomaterials with cells. Unlike mechanical heart valves, engineered biomimetic valves could develop and grow with the recipients. Such tissues and organs could someday be manufactured with a 3D printer, with the right bio-ink such as that developed by Savoji and his colleagues.

Houman Savoji and Arman Jafari

Credit: CHU Sainte-Justine

"My team has shown that an ink composed of polyvinyl alcohol, gelatin and k-carrageenan can be used to print heart valves that open and close correctly and has in-vitro and in-vivo biocompatibility and anti-thrombogenic properties," he explained. "They function well in a physiological environment like that of the human body, in both adult and children's sizes."

This compound also provides a structure (called a "scaffold") in which stem cells can potentially grow until they are replaced by a fully living tissue. Better still, in laboratory tests the valves generated fewer adverse effects than the mechanical or animal valves currently used in patients.

"These results suggest that our valves may be associated with a lower risk of complications than those currently used in transplants," said Jafari. "And since these are biomimetic artificial tissues, they can potentially grow with a transplanted child, limiting the need for repeat surgery."

Over the next few years, the scientists plan to pursue their research through in vivo trials, with the ultimate goal of seeing this technology one day be made available for use in a real-life, surgical setting.

Credit: CHU Sainte-Justine

Credit: CHU Sainte-Justine

Credit: CHU Sainte-Justine

"Formulation and evaluation of PVA/gelatin/carrageenan inks for 3D printing and development of tissue-engineered heart valves," by Aman Jafari et al, was published Oct. 10, 2023 in Advanced Functional Material. Funding was provided by the Fonds de recherche du Qubec - Sant, the Natural Sciences and Engineering Research Council of Canada, the TransMedTech Institute, the CHU Sainte-Justine Research Centre and Universit de Montral. The Savoji lab also benefits from equipment funded by CHU Sainte-Justine Foundation. Arman Jafari also received a doctoral scholarship from the FRQS and a bursary of excellence from Universit de Montral's Faculty of Medicine.

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Major Breakthrough: UofM Research Team 3D Prints Heart Valves - Mirage News