Gene Therapy For Beta-Thalassemia: Updated Perspectives | TACG – Dove Medical Press

Garyfalia Karponi,1,* Nikolaos Zogas2,*

1Department of Veterinary Medicine, Aristotle University of Thessaloniki, Thessaloniki, Greece; 2Department of Biology, Aristotle University of Thessaloniki, Thessaloniki, Greece

*These authors contributed equally to this work

Correspondence: Garyfalia KarponiLaboratory of Microbiology and Infectious Diseases, Faculty of Veterinary Medicine, Aristotle University of Thessaloniki, Thessaloniki 54124, GreeceTel +30 2310-999-956Fax +30 2310-999-934Email gkarponi@vet.auth.gr

Abstract: Allogeneic hematopoietic stem cell transplantation was until very recently, the only permanent curative option available for patients suffering from transfusion-dependent beta-thalassemia. Gene therapy, by autologous transplantation of genetically modified hematopoietic stem cells, currently represents a novel therapeutic promise, after many years of extensive preclinical research for the optimization of gene transfer protocols. Nowadays, clinical trials being held on a worldwide setting, have demonstrated that, by re-establishing effective hemoglobin production, patients may be rendered transfusion- and chelation-independent and evade the immunological complications that normally accompany allogeneic hematopoietic stem cell transplantation. The present review will offer a retrospective scope of the long way paved towards successful implementation of gene therapy for beta-thalassemia, and will pinpoint the latest strategies employed to increase globin expression that extend beyond the classic transgene addition perspective. A thorough search was performed using Pubmed in order to identify studies that provide a proof of principle on the aforementioned topic at a preclinical and clinical level. Inclusion criteria also regarded gene transfer technologies of the past two decades, as well as publications outlining the pitfalls that precluded earlier successful implementation of gene therapy for beta-thalassemia. Overall, after decades of research, that included both successes and pitfalls, the path towards a permanent, donor-irrespective cure for beta-thalassemia patients is steadily becoming a realistic approach.

Keywords: gene therapy, gene editing, thalassemia, mobilization, viral vectors, clinical trials, hematopoietic stem cells

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Adipose Tissue-derived Stem Cells Market to Witness Comprehensive Growth by 2018 2028 – Commerce Gazette

Adipose tissue is rich in multi potent stem cells that have the capability to differentiate into a number of cell types including adipocytes, osteocytes, chondrocytes and others, in vitro. These Adipose Tissue-derived Stem Cells are used for a number of applications including stem cell differentiation studies, regenerative medicine, cell therapy, tissue engineering and development of induced pluripotent stem cell lineage. Adult stem cells such as the Adipose Tissue-derived Stem Cells have a very good potential for regenerative medicine. The Adipose Tissue-derived Stem Cells show higher yields compared with other stem cell sources. Some of the regenerative medicine applications using Adipose Tissue-derived Stem Cells include skin, bone and cartilage regeneration.

Although, Adipose Tissue-derived Stem Cells have the ability to differentiate into different cell types in vitro, unlike the embryonic stem cells they lack the ability to differentiate into all types of organs and tissues of the body. Derivation of stem cells from adipose tissue have a number of advantages including that fat tissue contain 100 to 1000 times more mesenchymal stem cells than the bone marrow. Furthermore the method of collection of fat tissue is relatively easier and is less invasive than that of bone marrow collection. Although Adipose Tissue-derived Stem Cells have a potential to be used in cell-based therapy, there are a number of challenges the Adipose Tissue-derived Stem Cells market has to face. Some of the challenge include the safety issue for the clinical use of Adipose Tissue-derived Stem Cells, development and differentiation of the cells, delivery of the cells in vivo and immune response after the transplant.

The global Adipose Tissue-derived Stem Cells market is segmented based on product type and end user. Based on product type the Adipose Tissue-derived Stem Cells can be categorized into cell line and reagent & kits. Cell line can be further classified based on the source of the adipose tissue such as human and rodents. Based on reagents the Adipose Tissue-derived Stem Cells market is further classified as media & sera and kits. Based on application the Adipose Tissue-derived Stem Cells market is classified into regenerative medicine, cell therapy, tissue engineering, and other applications such as cell differentiation studies and other similar research. End users of Adipose Tissue-derived Stem Cells market are biotechnology companies and academic and research institutes.

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The Global Adipose Tissue-derived Stem Cells market is classified on the basis of product type, end user and region:

Based on the Product Type, Adipose Tissue-derived Stem Cells market is segmented into following: Cell Line Human Adipose Tissue-derived Stem Cells Rat Adipose Tissue-derived Stem Cells Reagents & Kits Media & Sera Kits

Based on the application, Adipose Tissue-derived Stem Cells market is segmented into following: Regenerative medicine Tissue engineering Cell therapy Others

Based on the end user, Adipose Tissue-derived Stem Cells market is segment as below: Biotechnology companies Academic & Research Institutes

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Growing research activities using stem cells along with the growth of regenerative medicine and cell therapy the global Adipose Tissue-derived Stem Cells market is set to expand considerably during the forecast period. However, ethical concerns and stringent regulations may hinder the growth of the global Adipose Tissue-derived Stem Cells market.

On the basis of geography, global Adipose Tissue-derived Stem Cells market is segmented into six major regions that include North America, Latin America, Europe, Asia-Pacific excluding China, China and Middle East & Africa. North America is expected to be the most lucrative Adipose Tissue-derived Stem Cells market owing to increased research activity of stem cells. Furthermore government support for regenerative and stem cell based studies along with cell therapy studies is driving the growth of the Adipose Tissue-derived Stem Cells market in the region. Changing government regulations in china is supporting the research activity that supports the growth of the adipose tissue-derived stem cell market in the region at a considerable rate.

Key participants operating in the Adipose Tissue-derived Stem Cells market are: Lonza, ThermoFisher Scientific, Celprogen, Inc, American CryoStem, Rexgenero Ltd, iXCells Biotechnologies, Merck KGaA, Lifeline Cell Technology, and others.

The report covers exhaustive analysis on: Adipose Tissue-derived Stem Cells Market Segments Adipose Tissue-derived Stem Cells Market Dynamics Historical Actual Market Size, 2013 2017 Adipose Tissue-derived Stem Cells Market Size & Forecast 2018 to 2026 Adipose Tissue-derived Stem Cells market Current Trends/Issues/Challenges Competition & Companies involved Adipose Tissue-derived Stem Cells Market Drivers and Restraints

Regional analysis includes North America Latin America Europe Asia Pacific Excluding China China Middle East & Africa

Report Highlights: Shifting Industry dynamics In-depth market segmentation Historical, current and projected industry size recent industry trends Key Competition landscape Strategies for key players and product offerings Potential and niche segments/regions exhibiting promising growth A neutral perspective towards market performance

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Adipose Tissue-derived Stem Cells Market to Witness Comprehensive Growth by 2018 2028 - Commerce Gazette

Suntanner, Heal Thyself: Exosome Therapy May Enable Better Repair of Sun, Age-Damaged Skin – NC State News

In the future, you could be your very own fountain of youth or at least your own skin repair reservoir. In a proof-of-concept study, researchers from North Carolina State University have shown that exosomes harvested from human skin cells are more effective at repairing sun-damaged skin cells in mice than popular retinol or stem cell-based treatments currently in use. Additionally, the nanometer-sized exosomes can be delivered to the target cells via needle-free injections.

Exosomes are tiny sacs (30 150 nanometers across) that are excreted and taken up by cells. They can transfer DNA, RNA or proteins from cell to cell, affecting the function of the recipient cell. In the regenerative medicine field, exosomes are being tested as carriers of stem cell-based treatments for diseases ranging from heart disease to respiratory disorders.

Think of an exosome as an envelope with instructions inside like one cell mailing a letter to another cell and telling it what to do, says Ke Cheng, professor of molecular biomedical sciences at NCState, professor in the NCState/UNC-Chapel Hill Joint Department of Biomedical Engineering and corresponding author of a paper describing the work. In this case, the envelope contains microRNA, non-coding RNA that instructs the recipient cell to produce more collagen.

To test whether exosomes could be effective for skin repair, Cheng and his team first grew and harvested exosomes from skin cells. They used commercially available human dermal fibroblast cells, expanding them in a suspension culture that allowed the cells to adhere to one another, forming spheroids. The spheroids then excreted exosomes into the media.

These 3D structures generate more procollagen more potent exosomes than you get with 2D cell expansion, says Cheng.

In a photoaged, nude mouse model, Cheng tested the 3D spheroid-grown exosomes against three other treatments: retinoid cream; 2D-grown exosomes; and bone marrow derived mesenchymal stem cells (MSCs) exosomes, a popular stem cell-based anti-aging treatment currently in use. The team compared improvements in skin thickness and collagen production after treatment. They found that skin thickness in 3D exosome treated mice was 20% better than in the untreated and 5% better than in the MSC-treated mouse. Additionally, they found 30% more collagen production in skin treated with the 3D exosomes than in the MSC treated skin, which was the second most effective treatment.

I think this study shows the potential for 3D exosomes to be used in anti-aging skin treatments, says Cheng. There are two major benefits to exosome treatments over conventional treatments: one, you can use donor skin cells from anyone to grow and harvest these exosomes they arent cells, so you dont run the risk of rejection. And two, the treatment can be administered without needles exosomes are small enough to be able to penetrate the skin via pressure, or jet injection methods.

Our hope is that eventually people may be able to bank skin samples and come back to them, or use donor exosome treatments that they can administer themselves. We believe that this work is an important step toward potentiating future human clinical trials in the prevention and treatment of cutaneous aging.

The work appears in ACS Nano, and was sponsored in part by the National Institutes of Health and the American Heart Association. Postdoctoral researcher Shiqui Hu is first author.

-peake-

Note to editors: An abstract follows.

Needle-Free Injection of Exosomes Derived from Human Dermal Fibroblast Spheroids Ameliorates Skin Photoaging

DOI: 10.1021/acsnano.9b04384

Authors: Shiqi Hu, Zhenhua Li, Jhon Cores, Ke Huang, Teng Su, Phuong-Uyen Dinh, Ke Cheng, North Carolina State University Molecular Biomedical Sciences Department and NCState/UNC-Chapel Hill Joint Department of Biomedical EngineeringPublished: Online in ACS Nano

Abstract: Human dermal fibroblasts (HDFs), the main cell population of the dermis, gradually lose their ability to produce collagen and renew intercellular matrix with aging. One clinical application for the autologous trans-dermis injection of HDFs that has been approved by the Food and Drug Administration aims to refine facial contours and slow down skin aging. However, the autologous HDFs used vary in quality according to the state of patients and due to many passages they undergo during expansion. In this study, factors and exosomes derived from three-dimensional spheroids (3D HDF-XOs) and the monolayer culture of HDFs (2D HDF-XOs) were collected and compared. 3D HDF-XOs expressed a significantly higher level of tissue inhibitor of metalloproteinases-1 (TIMP-1) and differentially expressed miRNA cargos compared with 2D HDF-XOs. Next, the efficacy of 3D HDF-XOs in inducing collagen synthesis and antiaging was demonstrated in vitro and in a nude mouse photoaging model. A needle-free injector was used to administer exosome treatments. 3D HDF-XOs caused increased procollagen type I expression and a significant decrease in MMP-1 expression, mainly through the downregulation of tumor necrosis factor-alpha (TNF-) and the upregulation of transforming growth factor beta (TGF-). In addition, the 3D-HDF-XOs group showed a higher level of dermal collagen deposition than bone marrow mesenchymal stem cell-derived exosomes. These results indicate that exosomes from 3D cultured HDF spheroids have anti-skin-aging properties and the potential to prevent and treat cutaneous aging.

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Suntanner, Heal Thyself: Exosome Therapy May Enable Better Repair of Sun, Age-Damaged Skin - NC State News

Brain tumors form synapses with healthy neurons, Stanford-led study finds – Stanford Medical Center Report

Using optogenetic techniques, which relied on laser light to activate the cancer cells in mice implanted with human gliomas, the researchers demonstrated that increasing electrical signals into the tumors caused more tumor growth. Proliferation of the tumors was largely prevented when glioma cells expressed a gene that blocked transmission of the electrical signals.

Existing drugs that block electrical currents also reduced growth of high-grade gliomas, the research found. A seizure medication called perampanel, which blocks activity of neurotransmitter receptors on the receiving end of a synapse, reduced proliferation of pediatric gliomas implanted into mice by 50%. Meclofenamate, a drug that blocks the action of gap junctions, resulted in a similar decrease in tumor proliferation.

Monjes team plans to continue investigating whether blocking electrical signaling within tumors could help people with high-grade gliomas. Its a really hopeful new direction, and as a clinician Im quite excited about it, she said.

Other Stanford co-authors of the paper are staff scientist Wade Morishita, PhD; postdoctoral scholars Anna Geraghty, PhD, Marlene Arzt, MD, and Kathryn Taylor, PhD; graduate student Shawn Gillespie; medical student Lydia Tam; staff scientist Cedric Espenel, PhD; research assistants Anitha Ponnuswami, Lijun Ni and Pamelyn Woo; Hannes Vogel, MD, professor of pathology and of pediatrics; and Robert Malenka, MD, PhD, professor of psychiatry and behavioral sciences.

Monje is a member of Stanford Bio-X, the Stanford Institute for Stem Cell Biology and Regenerative Medicine, the Stanford Maternal & Child Health Research Institute, the Stanford Cancer Institute and the Wu Tsai Neurosciences Institute at Stanford.

Scientists from Massachusetts General Hospital, Harvard Medical School, the Massachusetts Institute of Technology, Johns Hopkins University, the University of Michigan and the University of California-San Francisco also contributed to the research.

The research was funded by the National Institutes of Health (grant DP1 NS111132), the National Institute of Neurological Disorders and Stroke (grant NINDS R01NS092597), the National Cancer Institute (grant F31CA200273), the Michael Mosier Defeat DIPG Foundation, the ChadTough Foundation, the V Foundation, Ians Friends Foundation, the Department of Defense, the Mckenna Claire Foundation, Alexs Lemonade Stand Foundation, The Cure Starts Now Foundation and DIPG Collaborative, the Lyla Nsouli Foundation, Unravel Pediatric Cancer, the California Institute for Regenerative Medicine, the Joey Fabus Childhood Cancer Foundation, the N8 Foundation, the Sam Jeffers Foundation, Cancer Research UK, the Virginia and D.K. Ludwig Fund for Cancer Research, and the Stanford Maternal & Child Health Research Institutes Anne T. and Robert M. Bass Endowed Faculty Scholarship in Pediatric Cancer and Blood Diseases.

Stanfords Department of Neurology and Neurological Sciences also supported the work.

A second paper showing similar findings by another team of researchers was published simultaneously in Nature.

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Brain tumors form synapses with healthy neurons, Stanford-led study finds - Stanford Medical Center Report

Stem Cell Assay Market Expected to Secure Notable Revenue Share During 2019-2027 – Commerce Gazette

Regenerative medicine has emerged as new paradigm in human health. It has the potential to resolve unmet medical needs. Rapid growth in the interdisciplinary field of regenerative medicine is altering the health care domain by converting fundamental science into a variety of regenerative technologies. Stem cell is an undifferentiated mass of cell that has the ability to divide indefinite times. It can be further differentiated into specialized cells such as blood cells, skin cells, neurons, heart cells, chondrocytes, and osteocytes under specific conditions. Unspecialized nature, self-renewal capability, and dedifferentiation are the unique features of stem cells. Thus, these cells are useful in different applications in pharmaceutical research and medical fields. Stem cell research has grown significantly since 1978, when stem cells were discovered in human cord blood. Incidence of cancer is increasing across the globe due to the rise in aging population and changing lifestyle habits. This, in turn, is boosting the demand for anticancer drugs and therapies. According to the Centers for Disease Control and Prevention, 14.1 million new cancer cases were diagnosed around the globe in 2012 and around 19.3 million new cancer cases are expected to be diagnosed each year by 2025. Rise in incidences of chronic diseases is boosting the demand for research, making stem cells a highly preferred system for drug discovery due to its self-renewal capability and unspecialized nature.

Over the last decade, the application of cell-based assays has increased at a rapid pace among research institutes and pharmaceutical industries. This was primarily ascribed to the ethical issues associated with the use of animals for clinical trials. Furthermore, rise in approvals of clinical trials for stem cells based therapy, increase in funds from government organizations, and technological advancements are some of the factors driving the stem cell assay market. But, human embryonic stem cells, which are derived from inner cell mass of blastocyst are currently high on the political issues ethical concerns in many countries hampering the growth of the market. Additionally, lack of required infrastructure in developing countries and high cost associated with products are some of the factors restraining the stem cell assay market. Evolution of new therapies and low regulatory frameworks in emerging regions are expected to provide opportunities for market growth during the forecast period.

The global stem cell assay market has been segmented based on product, assay type, application, end-user, and region. In terms of product, the market for stem cell assay has been divided into human embryonic stem cell kits and adult stem cell kits. The adult stem cell kits segment is further divided into induced pluripotent stem cells kits, hematopoietic stem cell kits, mesenchymal stem cell kits, umbilical cord stem cell kits, and others. The adult stem cell kits segment is expected to account for the prominent share of the global stem cell assay market during the forecast period, led by the rise in product innovation activities and increasing focus on drug screening by biotechnology and pharmaceutical industries. Based on assay, the global stem cell assay market has been segregated into viability or cytotoxicity assay, cell identification assay, proliferation assay, differentiation assay, apoptosis assay, isolation & purification assay, and functional assay. Among these, the viability or cytotoxicity assay segment is anticipated to constitute key share of the global stem cell assay market during the forecast period, as cytotoxicity is an unavoidable stage during research.

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In terms of application, the global stem cell assay market has been segmented into drug discovery & development, regenerative medicine & therapy development, and clinical research. The regenerative medicine & therapy development segment is anticipated to expand at a rapid pace during the forecast period due to the rise in incidence of Parkinsons, Alzheimers, diabetes, and cancer diseases. This is anticipated to augment the focus on the development of new therapies and innovative drugs. Evolution of new therapies is estimated to provide new opportunities for the growth of the stem cell assay market during the forecast period.

Based in end-user, the global stem cell assay market has been segregated into government research institutes, private research institutes, and industry research. The industry research segment is projected to account for the major share of the global stem cell assay market during the forecast period. Growth in adoption of stem cell assays for drug screening process and testing is likely to drive the segment in the near future.

In terms of geography, the global stem cell assay market has been divided into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is expected to dominate the global stem cell assay market during the forecast period. Governmental initiatives for stem cell based research in North America are anticipated to boost the stem cell assay market in the region. The stem cell assay market in Asia Pacific is estimated to expand at a rapid pace; it is projected to overtake Europe in the near future. Development in the clinical research field and rise in patient pool are projected to augment the adoption of stem cell assay in Asia Pacific.

Key players operating in the stem cell assay market are Thermo Fisher Scientific,Merck KGaA, Promega Corporation, STEMCELL Technologies Inc., Bio-Techne Corporation, GE Healthcare, Cellular Dynamics International Inc., Hemogenix, Bio-Rad Laboratories, Inc., and Cell Biolabs Inc.

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The report offers a comprehensive evaluation of the market. It does so via in-depth qualitative insights, historical data, and verifiable projections about market size. The projections featured in the report have been derived using proven research methodologies and assumptions. By doing so, the research report serves as a repository of analysis and information for every facet of the market, including but not limited to: Regional markets, technology, types, and applications.

The study is a source of reliable data on: Market segments and sub-segments Market trends and dynamics Supply and demand Market size Current trends/opportunities/challenges Competitive landscape Technological breakthroughs Value chain and stakeholder analysis

The regional analysis covers: North America (U.S. and Canada) Latin America (Mexico, Brazil, Peru, Chile, and others) Western Europe (Germany, U.K., France, Spain, Italy, Nordic countries, Belgium, Netherlands, and Luxembourg) Eastern Europe (Poland and Russia) Asia Pacific (China, India, Japan, ASEAN, Australia, and New Zealand) Middle East and Africa (GCC, Southern Africa, and North Africa)

The report has been compiled through extensive primary research (through interviews, surveys, and observations of seasoned analysts) and secondary research (which entails reputable paid sources, trade journals, and industry body databases). The report also features a complete qualitative and quantitative assessment by analyzing data gathered from industry analysts and market participants across key points in the industrys value chain.

A separate analysis of prevailing trends in the parent market, macro- and micro-economic indicators, and regulations and mandates is included under the purview of the study. By doing so, the report projects the attractiveness of each major segment over the forecast period.

Highlights of the report: A complete backdrop analysis, which includes an assessment of the parent market Important changes in market dynamics Market segmentation up to the second or third level Historical, current, and projected size of the market from the standpoint of both value and volume Reporting and evaluation of recent industry developments Market shares and strategies of key players Emerging niche segments and regional markets An objective assessment of the trajectory of the market Recommendations to companies for strengthening their foothold in the market

Note:Although care has been taken to maintain the highest levels of accuracy in TMRs reports, recent market/vendor-specific changes may take time to reflect in the analysis.

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Global Bio-Banks Market : Industry Analysis and Forecast (2017-2026) By Type, Product,Application,Analysis,and Region. – OnYourDesks

Global Bio-Banks Market was valued US$ 2.3 Bn in 2017 and is expected to reach US$ 6.7 Bn by 2026, at CAGR of 14.3% during forecast period.

Global Bio-Banks MarketAdvancement in biobanking operations in order to ensure optimization of sample storage and maintenance is one of the key factors driving this market. Regenerative medicine through stem cell technology is one of the important treatments for diseases, like Alzheimers, diabetes, cancers, and rare genetic diseases. In order to benefit from the existing therapies, umbilical cord cells and other stem cells are preserved. With the increase in awareness about stem cell therapies, there has been a larger number of parents who are choosing umbilical cord banks for their children. There are a number of donor banks that are coming up as well. Biobanks not only support in the therapies for genetic diseases but also in medical research on rare genetic disorders. Growing awareness about stem cell therapies and innovation in the field of regenerative medicine are driving the growth of the global biobank market.

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Growing in the incidence of chronic diseases, government initiatives, development in drug discovery, and innovation of regenerative medicines, increasing healthcare expenditure and improvement in the treatment of cell and tissue disorders are some of the key factors boosting the global biobanks market. Furthermore, increasing awareness about biobanks is projected to boost the market for biobanks. Rising demographics, economies, and growth in GDP in the emerging countries like India and China, technological advancement and new innovate techniques are expected to offer good opportunities in the global biobanks market. Green banking and virtual biobanks for energy efficiency are some of the key trends that have been observed in global biobanks market. At the same time, expensive techniques, lack of standardization, economic recession and ethical issues related are some of the major factors limiting the growth for global biobanks market.

According to various application, the biobank application is expected to hold a XX% share during the forecast period. On account of different biospecimens stored at biobanks are witnessing significant demand because to advancements in cell-based research activities. Growing demand from different end users has led to the establishment of a substantial number of population-based and disease-based banks in a few years. Population-based banks are established to support precision medicine research initiatives, whereas disease-specific biobanks provide resources to research communities to enable a better understanding of disease etiology.

Among the regions, North America presently leads the global market for biobanks, closely followed by Europe, and it is expected to expand further at the highest CAGR during the forecast period. The increasing demand for Bio-Banks in the U.S. and Canada has allowed the province to have the highest market share. The prominence of these regions on account of the increasing incidence of chronic diseases, the imperative need to find effective treatments for them, large amounts of government investments in the area of biobanks, and the growing number of research activities, together with drug discovery in the region.

The Bio-Banks market report contains in-depth analysis of major drivers, opportunities, challenges, industry trends and their impact on the market. The Bio-Banks market report also provides data about the company and its strategy. This report also provides information on the competitive landscape section of the report provides a clear insight into the market share analysis of key industry players. This research report also adds a snapshot of key competition, market trends during the forecast period, expected growth rates and the primary factors driving and impacting growth market data. This information will be beneficial or helpful to the decision makers.

The objective of the report is to present a comprehensive assessment of the market and contains thoughtful insights, facts, historical data, industry-validated market data and projections with a suitable set of assumptions and methodology. The report also helps in understanding the global Bio-Banks market dynamics, structure by identifying and analysing the market segments and project the global market size. Further, the report also focuses on the competitive analysis of key players by product, price, financial position, product portfolio, growth strategies, and regional presence. The report also provides PEST analysis, PORTERs analysis, and SWOT analysis to address questions of shareholders to prioritizing the efforts and investment in the near future to the emerging segment in the global Bio-Banks market.

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Scope of Global Bio-Banks Market:

Global Bio-Banks Market, ByType:

Optimized Pre-Formulated Media Non-Optimized, Isotonic Formulation MediaGlobal Bio-Banks Market, By Product:

Refrigerators Ice Machines Freezers LN2 Supply Tanks Alarm and Monitoring Systems Cryogenic Storage Systems AccessoriesGlobal Bio-Banks Market, By Application:

Biobanking Regenerative Medicine Drug DiscoveryGlobal Bio-Banks Market, By Analysis:

Human tissue and tumor cells Bio-fluids Stem cells Umbilical cordGlobal Bio-Banks Market, By Region:

North America Europe Asia-Pacific South America Middle East & AfricaKey Players Operated in Market Include:

Home-Brew media solutions Teva Pharmaceuticals Organ Recovery Systems Genzyme Thermofisher Scientific VWR International Beckman Coulter Inc. Taylor-Wharton Tecan AG Panasonic Biomedical Sales Europe B.V. Thermo Fisher Scientific Inc. Taylor-Wharton International LLC So-Low Environmental Equipment Co.

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MAJOR TOC OF THE REPORT

Chapter One: Bio-Banks Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Bio-Banks Market Competition, by Players

Chapter Four: Global Bio-Banks Market Size by Regions

Chapter Five: North America Bio-Banks Revenue by Countries

Chapter Six: Europe Bio-Banks Revenue by Countries

Chapter Seven: Asia-Pacific Bio-Banks Revenue by Countries

Chapter Eight: South America Bio-Banks Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Bio-Banks by Countries

Chapter Ten: Global Bio-Banks Market Segment by Type

Chapter Eleven: Global Bio-Banks Market Segment by Application

Chapter Twelve: Global Bio-Banks Market Size Forecast (2019-2026)

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Global Bio-Banks Market : Industry Analysis and Forecast (2017-2026) By Type, Product,Application,Analysis,and Region. - OnYourDesks

Translational Regenerative Medicine Market : 2024 Shares, Trend and Growth – Bee Tribune

Regenerative medicine is a segment of translational research in molecular biology and tissue engineering. It involves the process of regeneration of human cells, tissues, or organs to re-establish their normal functions through stimulation of bodys repair system. They are widely used in the treatment of many degenerative disorders occurring in the areas of dermatology, orthopedic, cardiovascular and neurodegenerative diseases. Stem cell therapy is the available tool in the field of translational regenerative medicine. It has gained importance in the past few years as it is a bio-based alternative to synthetic options. Stem cells have high power of regeneration. Hence, these enable production of other cells in the body. This has increased demand for stem cell therapy in the treatment of degenerative diseases. Currently, stem cell therapy has applications in the treatment of diseases such as autism, cancer, retinal diseases, heart failure, diabetes, rheumatoid arthritis, Alzheimers. Extensive research is being carried out on stem cell therapy. The Centre for Commercialization of Regenerative Medicine (CCRM) has reported around 1900 active clinical trials undergoing currently. It also reported 574 active industry-sponsored cell therapy clinical studies, 50 of these are in phase 3 development. Hence, stem cell therapy is projected to contribute to the growth of the translational regenerative medicine market. However, ethical issues in the use of embryonic stem cells is likely to restrain the market.

Rising prevalence of degenerative diseases, aging population, rapid growth of emerging countries, and technical advancements in developed countries are the major factors fueling the growth of the translational regenerative medicine market.

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The global translational regenerative medicine market has been segmented based on product type, therapy, application, and region. In terms of product type, the market has been categorized into cellular and acellular. The cellular segment dominated the global market in 2016. Based on therapy, the global translational regenerative market has been segmented into cell therapy, gene therapy, immunotherapy, and tissue engineering. Immunotherapy is projected to be the fastest growing segment during the forecast period. In terms of application, the market has been segmented into orthopedic & musculoskeletal, cardiology, diabetes, central nervous system diseases, dermatology, and others. Cardiology and orthopedic & musculoskeletal are anticipated to be the fastest growing segments of the global translational regenerative medicine market.In terms of region, the global translational regenerative medicine market has been segmented into North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. North America dominated the global regenerative medicine market owing to a large number of leading companies and expansion of research and development activities in the U.S. Increased medical reimbursement and advanced health care also drive the market in the region. Orthopedic is the leading application segment contributing to the growth of the market in the region. Asia Pacific is forecasted the huge growth because of large consumer pool, rising income, and health care expenditure. However, the market in Asia Pacific could face challenges such as high cost of bio-based medicines and stringent regulatory policies.

The global translational regenerative medicine market is dominated by key players such as CONMED Corporation, Arthrex, Inc., Organogenesis, Inc., Nuvasive, Inc., Osiris Therapeutics, Inc., Celgene Corporation, Brainstorm Cell Therapeutics Inc. and Medtronic.

The report offers a comprehensive evaluation of the market. It does so via in-depth qualitative insights, historical data, and verifiable projections about market size. The projections featured in the report have been derived using proven research methodologies and assumptions. By doing so, the research report serves as a repository of analysis and information for every facet of the market, including but not limited to: Regional markets, technology, types, and applications.

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Translational Regenerative Medicine Market : 2024 Shares, Trend and Growth - Bee Tribune

Greek Woman’s Claim to Be Scientist Refuted by NASA – The National Herald

By TNH Staff September 21, 2019

(NASA, ESA, S. Beckwith (STScI), HUDF Team via AP)

A 31-year-old Greek woman who has racked up international acclaim as a renowned scientist isnt one, academics said, and the US space agency NASA said she never worked there as she claimed.

Eleni Antoniadou, 31, has won praise and awards around the world for her supposed work in a wide range of fields, including regenerative medicine, artificial organ bioengineering and space medicine at NASA.

The British newspaper The Telegraph said it was told by NASA she had not been on the staff there and university professors also disputed her assertions, undercutting her frequent appearances n the media and her claims for international achievements and as she was just presented an achievement award by Greek Education Minister Niki Kerameus who said, Her passion for science inspires us and fills us with optimism.

A Facebook post by Costas Bouyioukos, assistant professor of bioinformatics at Paris Diderot University in France that went viral went even further in discrediting her as he said She is not even fit to be called a scientist for most people.

Bouyioukos said she only and only completed the space agencys Frontier Development Lab, an eight-week educational program.

Antoniadou, the inspiration for Greeces first Barbie doll, has been described as a specialist in the fields of regenerative medicine, artificial organ bioengineering and space medicine, as well as training astronauts at NASA, and working as CEO of Transplants Without Donors, which creates artificial organs for transplants, the paper said.

She has been called a Greek scientist of global calibre by Greek media and was voted 2013 Woman of the Year at the annual British FDM Everywoman in Technology Awards, winning the NASA-ESA Outstanding Researcher Award in 2012 which doesnt appear to be real and presiding over the European Health Parliament.

Greek Hoaxes, a team which debunks fake news, also dismissed her claim to have worked on a team that built the first trachea implant to be successfully used on a patient at University College London, saying the patient died afterwards, the paper reported.

She issued a statement on Facebook saying she was working on a project on artificial intelligence for NASA but would not comment to the paper while a spokesperson for the agency said she was not an employee there but couldnt say if she had worked as a sub-contractor on projects.

The National Herald earlier wrote of her achievements as well, saying that he had said of her motivation: Love another person, even when they lose themselves, when their hygiene is failing, when they dont eat, when they dont care if they are in the light or the darkness. When they have given up and you want to give them a kick and put them to bed. Love, even when youre not sure its worth it. Admire them, even if they look at you without actually seeing you.

She had said she was a researcher in the interdisciplinary fields of regenerative medicine and bioastronautics, specializing in the regeneration of artificial organs from stem cells as an alternative therapeutic pathway for transplants and worked on the creation of cerebral implants, artificial skin, muscles, ears, nerves and the esophagus.

She said she designed a series of bioreactors and tissue engineering tools and has created the worlds first amniotic fluid stem cell bank, conducted experimental studies on the development of bio-nanotubes as drug carriers for targeted cancer therapies, as well as clinical trials for stem cell therapies for lung cancer.

Antoniadou also said she was on the Advisory Committee of the Research and Analysis Organization, DIANEOSIS, in Greece and had been honored by the European Patent Organization in Germany but there were no reports in the wake of others challenging her background whether any of what she said she had done was true.

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Greek Woman's Claim to Be Scientist Refuted by NASA - The National Herald

Next generation sequencing for cancer precision medicine – Drug Target Review

The systematic successful treatment of cancer still eludes us and in an effort to refine this area of targeted medicine, Lauri Paasonen and colleagues explore the potential of using patient-derived cells (PDCs) for devising a personalised treatment strategy for solid tumours.

There are many successful cancer therapies on the market but still, depending on the cancer subtype and stage of the disease, many patients do not fully respond to treatment or can later present with a drug-resistant metastatic disease.

However, many new targeted therapies have been developed but their effectiveness for different cancer types has not been evaluated. In the precision medicine (PM) approach, a vast amount of clinical and omics data is collected from a patient in order to find potential drug targets and to translate the results back to the clinic. Importantly, this research may aid the understanding of drug resistance arising from inter/intratumour heterogeneity. While the genomic alterations, such as somatic driver mutations, have been thoroughly investigated, only a few can be targeted with drugs. This has led to the development of the functional PM approach, which has been successful, for example, in leukaemias. Here, the patient-derived cancer cells (PDCs) can be directly used without the need for cell culture steps for ex vivo drug sensitivity and resistance testing.1-3 The response of PDCs is tested against hundreds of approved and investigational drugs, depending on the platform. The combination of the drug response data with phenotypic and genotypic omics data and clinical information about the patient enables a personalised treatment strategy to be developed.

Applying the functional PM approach for solid tumours has been far more complicated compared with leukaemias and other haematologic cancers. This is mostly due to the challenges in growing primary PDCs ex vivo in an environment that allows them to sustain their molecular characteristics. Further development of culture conditions for PDCs grown two-dimensionally (2D) and threedimensionally (3D) as spheroid/organoid cultures has led to improved PDC models that match phenotypically, genotypically and functionally with the original cancer tissue.4-6 2D monolayer cultures of epithelial tumours can be efficiently used for functional high-throughput drug testing;5 however, the PDCs grown in 3D as organoids are generally considered to be a more physiologically relevant and geno- and phenotypically stable model for cancer.4 The optimisation of assays is important for studies with cancer organoids: for instance, they grow slowly, which may limit their use in highthroughput drug testing. Importantly, all types of PDCs cultured in the laboratory should be genetically characterised to confirm that they carry the same cancer driver mutations as the original tumour tissue from which they were derived. Ultimately, ex vivo drug testing with geneticallyand phenotypically-characterised PDCs from solid tumours allows us to tailor patient-specific treatments, to find new opportunities for drug repurposing and to gain relevant cell models for drug discovery.

Ex vivo drug testing with genetically- and phenotypically-characterised PDCs from solid tumours allows us to tailor patient-specific treatments

Next generation sequencing (NGS) has had a big impact on genomic research. High-throughput sequencing technologies have emerged that fulfil the need for sequencing of large gene quantities, such as the whole exome with ~23,000 different genes, and for the quantitative analysis of mutation frequencies. This has also enabled genome sequencing to be used as a clinical tool.7 The characterisation of somatic mutations and copy number variations in PDCs means they can be properly matched with the tumour tissue they are derived from and is a crucial step for any further PDC-based assay.8

Whole exome sequencing or targeted cancer panel sequencing can be performed for PDCs derived both from liquid biopsies and solid tumour samples, but the limited quantity of PDCs often sets certain requirements for the assays. In addition, the isolation of DNA of good quality and adequate quantity from solid tumour PDCs cultured in a 3D matrix can be challenging, as the matrix used may not be easy to remove or can cause steric hindrance during the isolation. Typically, the DNA isolation is performed using commercial column extraction methods, but when the cells are cultured in 3D, the properties of 3D matrices may not allow the proper filtration of samples through the DNA isolation column, resulting in an insufficient yield and poor DNA quality. However, by choosing a suitable 3D culture matrix and protocol, the isolation of DNA for NGS library construction can be performed.

Figure 1: The example images of renal cancer PDCs grown A) in 2D as a monolayer, and B) in 3D in GrowDex after cellulase enzyme treatment (spheroid diameter 216m). Samples were imaged with Leica S80 microscope, 10x objective.

PDCs cultured in 3D in GrowDex We have optimised the growth of renal PDCs in 2D as a monolayer and in 3D using different matrices (Figure 1). These matrices include GrowDex, a plant-based cellulose hydrogel that has recently been demonstrated as a suitable matrix for the 3D culturing of various cell types.9,10 It is an animal-free, clean and tunable hydrogel, which can be degraded using cellulase enzyme without impacting the human cells.9 As matrices used to support the growth of PDCs in 3D may hinder the typical column-based DNA purification prior to sequencing, we streamlined the workflow for whole exome sequencing (WES) from a low amount of renal cancer PDCs grown as spheroids in GrowDex. As a control, NGS was also performed for the cells cultured in a traditional 2D monolayer setup. The detailed protocol can be found as a separate application note.11

Shortly after, renal tumour tissue samples were dissociated and cultured in 2D according to previously published protocols12,13 or in 0.4 percent GrowDex hydrogel in complete growth medium in 24- or 96-well plates. GrowDex was degraded with cellulase enzyme GrowDase and DNA was isolated with Dynabeads DNA Direct kit. 150ng of DNA was used for library construction and exome sequencing was performed using Roche HyperCap MedExome. Resulting libraries were sequenced with Illumina Hiseq2500 Rapid PE100 runs. Sequence analysis and variant calling was performed using inhouse tools.14

We observed that removal of the 3D matrix is required for an efficient isolation of DNA from 3D-cultured cells by Dynabeads isolation kit. GrowDase enzyme treatment enabled the effective release of the PDC spheroids from GrowDex (Figure 1B) and the robust processing of the spheroids combined with Dynabeads on the magnetic rack. The success of exome capture is critically dependent on the amount and quality of input DNA. In general, a good yield of DNA was obtained from the PDCs cultured 3D in GrowDex; sufficient for WES or any other type of NGS assay. With the DNA isolated from 3D cultures, we were able to generate WES data with somatic resolution (mean target coverage >80x).11 The comparison of sequencing results at the chromosomal level with the Integrative Genomics Viewer15,16 revealed similar coverage in the sequencing of DNA isolated from PDCs cultured either traditionally in 2D monolayer on the plastic or 3D in GrowDex (Figure 2).

Figure 2: Integrative Genomics Viewer image shows the comparable sequencing coverage of TP53 for DNA isolated from 2D- and 3D-cultured PDCs.11

Novel 3D-culturing methods for PDCs enable more physiologically-relevant ex vivo cancer models. Fast and cost-effective genome sequencing of PDCs is needed for characterisation of the cells and the clinical implementation of ex vivo drug efficacy screening results in PM. Here, the renal cancer PDCs cultured in GrowDex in 3D were made accessible for lysis and bead-based DNA recovery with cellulase enzyme treatment degrading the nanofibrillar cellulose network. Additional purification steps typically present in DNA isolation were needed and the recovered amount and quality of DNA was sufficient for NGS library construction and exome sequencing. In conclusion, the development of fast, straightforward assay protocols optimised for 3D PDC spheroids/ organoids can further improve their utilisation for PM, drug discovery and other applications.

Lauri Paasonen is an application scientist in UPM Biomedicals, Helsinki, Finland focusing on 3D cell culture product and application development. He has a PhD in pharmaceutical sciences from the University of Helsinki, Finland. Before joining UPM in 2014, Lauri worked as a post-doctoral researcher at Sanford-Burnham Medical Research Institute, La Jolla, CA, USA and at the University of Helsinki, Finland.

Vilja Pietiinen is a senior scientist at the Institute for Molecular Medicine Finland (FIMM, HiLIFE, University of Helsinki) and a team leader of the personalised urological tumour project in Olli Kallioniemis research group. Her research focuses on systems medicine and precision therapeutics in cancer. She obtained an MSc in biochemistry and a PhD in virology/ cell biology in 2005 (University of Edinburgh, UK; University of Helsinki-UH, Finland). Currently, she is dedicated to developing high-content phenotypic imagebased characterisation and ex vivo drug testing of patientderived cancer cells.

Piia Mikkonen, Institute for Molecular Medicine Finland (FIMM), Pekka Ellonen, Helsinki Institute for Life Sciences (HiLIFE) and Aino Palva, University of Helsinki, Finland were also additional authors.

The authors would like to thank FIMM Genomics Core Unit (HiLIFE, University of Helsinki), funded by BioCenter Finland. The tissue samples were obtained through DEDUCER study (the Development of diagnostics and treatment of urological cancers; main investigator in the clinic: Associated Professor A Rannikko, University of Helsinki; with the approved study permissions for HUS/71/2017, 26.04.2017, ethical committee approval 15.03.2017 Dnro 154/13/03/02/2016, and patient consents). This work is based on research collaboration between academy and UPM, supported by UPM-Kymmene Corporation, Finland.

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Next generation sequencing for cancer precision medicine - Drug Target Review

Engineered killer T cells could provide long-lasting immunity against cancer – Mirage News

UCLA Broad Stem Cell Research Center

Lili Yang

Theyve been called the special forces of the immune system: invariant natural killer T cells. Although there are relatively few of them in the body, they are more powerful than many other immune cells.

In experiments with mice, UCLA researchers have shown they can harness the power of iNKT cells to attack tumor cells and treat cancer. The new method, described in the journal Cell Stem Cell, suppressed the growth of multiple types of human tumors that had been transplanted into the animals.

Whats really exciting is that we can give this treatment just once and it increases the number of iNKT cells to levels that can fight cancer for the lifetime of the animal, said Lili Yang, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA and the studys senior author.

Scientists have hypothesized that iNKT cells could be a useful weapon against cancer because it has been shown that they are capable of targeting many types of cancer at once a difference from most immune cells, which recognize and attack only one particular type of cancer cell at a time. But most people have very low quantities of iNKT cells; less than 0.1% of blood cells are iNKT cells in most cases.

Still, Yang and her colleagues knew that previous clinical studies have shown that cancer patients with naturally higher levels of iNKT cells generally live longer than those with lower levels of cells.

They are very powerful cells but theyre naturally present in such small numbers in the human blood that they usually cant make a therapeutic difference, said Yang, who also is a UCLA assistant professor of microbiology, immunology and molecular genetics and a member of the UCLA Jonsson Comprehensive Cancer Center.

The researchers goal was to create a therapy that would permanently boost the bodys ability to naturally produce more iNKT cells. They started with hematopoietic stem cells cells found in the bone marrow that can duplicate themselves and can become all types of blood and immune cells, including iNKT cells. The researchers genetically engineered the stem cells so that they were programmed to develop into iNKT cells.

They tested the resulting cells, called hematopoietic stem cell-engineered invariant natural killer T cells, or HSC-iNKT cells, on mice with both human bone marrow and human cancers either multiple myeloma (a blood cancer) or melanoma (a solid tumor cancer) and studied what happened to the mices immune systems, the cancers and the HSC-iNKT cells after they had integrated into the bone marrow.

They found that the stem cells differentiated normally into iNKT cells and continued to produce iNKT cells for the rest of the animals lives, which was generally about a year.

One advantage of this approach is that its a one-time cell therapy that can provide patients with a lifelong supply of iNKT cells, Yang said.

While mice without the engineered stem cell transplants had nearly undetectable levels of iNKT cells, in those that received engineered stem cell transplants, iNKT cells made up as much as 60% of the immune systems total T cell count. Plus, researchers found they could control those numbers by how they engineered the original hematopoietic stem cells.

Finally, the team found that in both multiple myeloma and melanoma, HSC-iNKT cells effectively suppressed tumor growth.

The studys co-first authors are Yanni Zhu, a UCLA project scientist, and Drake Smith, a UCLA doctoral student.

More work is needed to determine how HSC-iNKT cells might be useful for treating cancer in humans and whether increasing the number of HSC-iNKT cells could cause long-term side effects. But Yang said hematopoetic stem cells collected either from a person with cancer or a compatible donor could be used to engineer HSC-iNKT cells in the lab. The procedure for transplanting stem cells into patients bone marrow is already well-established as a treatment for many blood cancers.

Funding for the study was provided by the National Institutes of Health, the California Institute for Regenerative Medicine, the Concern Foundation, the STOP CANCER Foundation, a UCLA Broad Stem Cell Research Center Rose Hills Foundation Innovator Grant, and the centers training program, supported by the Sherry, Dave and Sheila Gold Foundation.

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Engineered killer T cells could provide long-lasting immunity against cancer - Mirage News