3D illustration of cells releasing exosomes
Scientists have developed a new gene-therapy technique by transforming human cells into mass producers of tiny nano-sized particles full of genetic material that has the potential to reverse disease processes.
Though the research was intended as a proof of concept, the experimental therapy slowed tumor growth and prolonged survival in mice with gliomas, which constitute about 80 percent of malignant brain tumors in humans.
The technique takes advantage of exosomes, fluid-filled sacs that cells release as a way to communicate with other cells.
While exosomes are gaining ground as biologically friendly carriers of therapeutic materials because there are a lot of them and they dont prompt an immune response the trick with gene therapy is finding a way to fit those comparatively large genetic instructions inside their tiny bodies on a scale that will have a therapeutic effect.
This new method relies on patented technology that prompts donated human cells such as adult stem cells to spit out millions of exosomes that, after being collected and purified, function as nanocarriers containing a drug. When they are injected into the bloodstream, they know exactly where in the body to find their target even if its in the brain.
Think of them like Christmas gifts: The gift is inside a wrapped container that is postage paid and ready to go, said senior study author L. James Lee, professor emeritus of chemical and biomolecular engineering at The Ohio State University.
And they are gifts that keep on giving, Lee noted: This is a Mother Nature-induced therapeutic nanoparticle.
The study is published in the journal Nature Biomedical Engineering.
In 2017, Lee and colleagues made waves with news of a regenerative medicine discovery called tissue nanotransfection (TNT). The technique uses a nanotechnology-based chip to deliver biological cargo directly into skin, an action that converts adult cells into any cell type of interest for treatment within a patients own body.
By looking further into the mechanism behind TNTs success, scientists in Lees lab discovered that exosomes were the secret to delivering regenerative goods to tissue far below the skins surface.
The technology was adapted in this study into a technique first author Zhaogang Yang, a former Ohio State postdoctoral researcher now at the University of Texas Southwestern Medical Center, termed cellular nanoporation.
The scientists placed about 1 million donated cells (such as mesenchymal cells collected from human fat) on a nano-engineered silicon wafer and used an electrical stimulus to inject synthetic DNA into the donor cells. As a result of this DNA force-feeding, as Lee described it, the cells need to eject unwanted material as part of DNA transcribed messenger RNA and repair holes that have been poked in their membranes.
They kill two birds with one stone: They fix the leakage to the cell membrane and dump the garbage out, Lee said. The garbage bag they throw out is the exosome. Whats expelled from the cell is our drug.
The electrical stimulation had a bonus effect of a thousand-fold increase of therapeutic genes in a large number of exosomes released by the cells, a sign that the technology is scalable to produce enough nanoparticles for use in humans.
Essential to any gene therapy, of course, is knowing what genes need to be delivered to fix a medical problem. For this work, the researchers chose to test the results on glioma brain tumors by delivering a gene called PTEN, a cancer-suppressor gene. Mutations of PTEN that turn off that suppression role can allow cancer cells to grow unchecked.
For Lee, founder of Ohio States Center for Affordable Nanoengineering of Polymeric Biomedical Devices, producing the gene is the easy part. The synthetic DNA force-fed to donor cells is copied into a new molecule consisting of messenger RNA, which contains the instructions needed to produce a specific protein. Each exosome bubble containing messenger RNA is transformed into a nanoparticle ready for transport, with no blood-brain barrier to worry about.
The advantage of this is there is no toxicity, nothing to provoke an immune response, said Lee, also a member of Ohio States Comprehensive Cancer Center. Exosomes go almost everywhere in the body, including passing the blood-brain barrier. Most drugs cant go to the brain.
We dont want the exosomes to go to the wrong place. Theyre programmed not only to kill cancer cells, but to know where to go to find the cancer cells. You dont want to kill the good guys.
The testing in mice showed the labeled exosomes were far more likely to travel to the brain tumors and slow their growth compared to substances used as controls.
Because of exosomes safe access to the brain, Lee said, this drug-delivery system has promise for future applications in neurological diseases such as Alzheimers and Parkinsons disease.
Hopefully, one day this can be used for medical needs, Lee said. Weve provided the method. If somebody knows what kind of gene combination can cure a certain disease but they need a therapy, here it is.
Read the original here:
A New Gene Therapy Strategy, Courtesy of Mother Nature - Global Health News Wire
- Century Therapeutics to Present at the 63rd American Society of Hematology Annual Meeting and Host Virtual Research & Development Update - Yahoo... - November 8th, 2021
- BioRestorative Therapies Prices $23 Million Public Offering - GlobeNewswire - November 8th, 2021
- Lab-Growing Everything Might Be The Only Way To Attain A Sustainable World - Intelligent Living - November 8th, 2021
- Probiotics Market to Experience Significant Growth during the Forecast Period 2021-2028 Bolivar Commercial - Bolivar Commercial - November 8th, 2021
- They demonstrate the existence of stem cells in the hippocampus of the human brain - Market Research Telecast - October 28th, 2021
- BioRestorative Therapies Announces Nomination of Two New Members to the Board of Directors - StreetInsider.com - October 28th, 2021
- Akiko Nishiyama Explains the Many Strengths of a Degree in Physiology and Neurobiology - UConn Today - UConn Today - October 28th, 2021
- New stem cell identified by Sanford Burnham Prebys researchers offers hope to people with rare liver disease - Newswise - October 16th, 2021
- The Impact Of Market Restrictions On The US Stem Cell Biomaterials Market - Med Device Online - October 16th, 2021
- College Student and Retired Teacher to Thank Stem Cell Donors They've Never Met for Saving Their Lives During City of Hope's 45th Bone Marrow... - October 16th, 2021
- Phase 2 Clinical Trial Data of NurOwn in Progressive MS Will Be Presented at the 37th Congress of the European Committee for Treatment and Research in... - October 16th, 2021
- Cell therapy strategies for COVID-19: Current approaches and potential applications - Science Advances - August 18th, 2021
- Stem Cell Therapy Market worth $40.3 billion by 2027 Exclusive Report by CoherentMarketInsights - PharmiWeb.com - August 18th, 2021
- Therapeutic Solutions International Reports Superior Neurogenesis Induction in Animal Model of Viral Induced Cognitive Dysfunction Compared to other... - August 18th, 2021
- Atf3 and Rab7b genes drive regeneration in mature cells - Baylor College of Medicine News - August 18th, 2021
- U of T's Medicine by Design helps unite international researchers working to map every human cell - News@UofT - August 18th, 2021
- Mesenchymal Stem Cells Market Witnesses Upward Trend with High Prevalence of Parkinson's Disease The Manomet Current - The Manomet Current - July 22nd, 2021
- BioRestorative Therapies Announces Notice of Allowance for a New Patent Application Related to its Off-the-Shelf ThermoStem Program -... - April 4th, 2021
- Transplant After CD19 CAR T-Cell Therapy Shows Durable Disease Control in Children, Young Adults With B-ALL - Cancer Network - April 4th, 2021
- Stem Cell Assay Market Changing Dynamics Of Competition With Forecast To 2030 The Bisouv Network - The Bisouv Network - April 4th, 2021
- Stem Cell Therapy Market Research Reveals Enhanced Growth During The Forecast Period 2017 2025 FLA News - FLA News - March 8th, 2021
- Adult Stem Cells Market Size, Share, Global Industry Overview, Challenges, Business Overview and Forecast Research Study 2024 (Effect of the COVID-19... - February 17th, 2021
- Be The Match encourages people of color to join bone marrow registry - KING5.com - February 17th, 2021
- Discovery for turning on cell repair in tissues and organs - Monash University - February 17th, 2021
- Manageable Safety Profile Observed in Phase 1 Studies Examining UCART19 for Pediatric and Adult Patients with B-Cell ALL - Cancer Network - February 11th, 2021
- The Very First Signs of an Immune Response Have Been Filmed in a Developing Embryo - ScienceAlert - February 11th, 2021
- JSP191 With Low Dose Irradiation and Fludarabine is Safe and Effective for Patients with MRD+ AML/MDS - Cancer Network - February 9th, 2021
- Creative Bioarray Offers Stem Cell Lines Generation Service for Promoting Scientific Research - Press Release - Digital Journal - February 9th, 2021
- JSP191 With Low Dose Irradiation and Chemotherpy Demonstrates Efficacy and Safety in MRD+ AML/MDS - Targeted Oncology - February 9th, 2021
- Stem Cells Market Value to Reach Around USD 17.79 Bn by 2027 - GlobeNewswire - February 4th, 2021
- Global Adult Stem Cells Market Revenue To Witness Humongous Elevation By 2026 Market Research Store Jumbo News - Jumbo News - February 4th, 2021
- Stem Cell Therapy Market to Score Past US$ 40.3 Billion Valuation by 2027: At a CAGR of 21.1% KSU | The Sentinel Newspaper - KSU | The Sentinel... - February 2nd, 2021
- Reduced Adult Neurogenesis Linked with Alzheimers Disease - The Scientist - February 2nd, 2021
- Regenerative Medicine (Bone and Joint) Market to Score Past US$ 14990.0 Million Valuation by 2027: CMI KSU | The Sentinel Newspaper - KSU | The... - February 2nd, 2021
- Orthopedic Regenerative Medicine Market Size to Witness A Lucrative Growth Over 2020-2027 | Curasan, Inc., Carmell Therapeutics Corporation, Anika... - February 2nd, 2021
- Adult Stem Cells Market Demand, Growth Challenges, Industry Analysis And Forecasts To 2026 | Epistem Ltd.,Globalstem,Mesoblast Ltd.,Brainstorm Cell... - February 2nd, 2021
- ToolGen ties up with 3D bioprinting company to apply induced pluripotent stem cells to gene correction - Aju Business Daily - February 2nd, 2021
- Medicine by Design researchers focus on promoting self-repair of the brain - News@UofT - January 31st, 2021
- Should You Buy Brainstorm Cell Therapeutics Inc (BCLI) Stock on Tuesday? - InvestorsObserver - January 31st, 2021
- Eye stem cell transplant to treat blindness bolsters retinal function in monkeys - FierceBiotech - January 14th, 2021
- Induction of muscle-regenerative multipotent stem cells from human adipocytes by PDGF-AB and 5-azacytidine - Science Advances - January 14th, 2021
- RoosterBio Partners with Sartorius to Advance Cell and Gene Therapy Manufacturing - GlobeNewswire - January 14th, 2021
- Reversing The Aging Clock With Epigenetic Reprogramming - Bio-IT World - January 14th, 2021
- New Models in Organoids Market Open New Vistas in Stem Cell Research for Cancer, Global Valuation to Reach US$ 12.8 Bn by 2030: TMR - PRNewswire - January 12th, 2021
- Health Canada Approves ONUREG (azacitidine tablets), First Maintenance Therapy for Patients in Remission from Acute Myeloid Leukemia - Canada NewsWire - January 12th, 2021
- Stem Cells Market 2020 Research Study including Growth Factors, Types and Application to 2026| Covid-19 Impact - Farming Sector - January 9th, 2021
- The real reason behind goosebumps - Jill Lopez - January 3rd, 2021
- Synthetic lethality across normal tissues is strongly associated with cancer risk, onset, and tumor suppressor specificity - Science Advances - January 3rd, 2021
- New Approaches to the Treatment of Relapsed or Refractory Diffuse Large B-cell Lymphoma - Targeted Oncology - January 3rd, 2021
- CAR T-Cell Therapies Are Set to Expand Into More Hematologic Malignancy Indications - Targeted Oncology - December 26th, 2020
- ElevateBio's HighPassBio Presents on Novel T Cell Receptor Cell Therapy for Leukemia Relapse at 62nd Annual ASH Meeting - Business Wire - December 6th, 2020
- CRISPR and another genetic strategy fix cell defects in two common blood disorders - Science Magazine - December 6th, 2020
- Multiple sclerosis iPS-derived oligodendroglia conserve their properties to functionally interact with axons and glia in vivo - Science Advances - December 6th, 2020
- UCART22 Safe and Active in CD22-Expressing B-Cell ALLs - Targeted Oncology - December 6th, 2020
- Cell Therapy Market Size, Share, Market Research and Industry Forecast Report, 2020-2027 (Includes Business Impact of COVID-19) - Cheshire Media - December 6th, 2020
- Stem Cells Market is Expected to Thrive at Impressive CAGR by 2025 - The Haitian-Caribbean News Network - December 5th, 2020
- Stem Cell Assay to Register Substantial Expansion by 2026| Merck, Thermo Fisher Scientific, GE Healthcare - The Haitian-Caribbean News Network - December 5th, 2020
- Stem Cell Therapy Global Market Report 2020-30: Covid 19 Growth and Change - GlobeNewswire - November 25th, 2020
- Functionally distinct resident macrophage subsets differentially shape responses to infection in the bladder - Science Advances - November 25th, 2020
- BioRestorative Therapies Emerges from Chapter 11 Reorganization Other OTC:BRTX - GlobeNewswire - November 20th, 2020
- Jakafi and Dacogen May Improve Overall Survival in Patients with MPN - Curetoday.com - November 20th, 2020
- BRTX-100; the Story of BioRestorative Therapies Inc (OTCMKTS: BRTX) - MicroCap Daily - November 20th, 2020
- As California Passes Prop 14, What Is Stem Cell Research and Why Is It Controversial? - Newsweek - November 16th, 2020
- Organoids mimic the early development of the heart in mouse embryos - BioNews - November 16th, 2020
- Human mesenchymal stromal cells do not express ACE2 and TMPRSS2 and are not permissive to SARS-CoV-2 infection - DocWire News - November 16th, 2020
- Cynata Therapeutics (ASX:CYP) begins phase three osteoarthritis trial - The Market Herald - November 16th, 2020
- $ 30.1 billion Worth Cell Expansion Market, Led by Thermo Fisher Scientific Inc, Becton, Dickinson and Company, Terumo BCT and Others. re:Jerusalem -... - November 3rd, 2020
- Impact of COVID-19 on Advanced Wound Care Management Market : Implications on Business Strategies, Countermeasures, Economic Impact - The Think... - November 1st, 2020
- Stem Cells Market 2020 Is Growing with Highest Size, Share of Top Key Players in the Industry and Forecast Survey Till 2024 - Zenit News - October 30th, 2020
- Somatic Mutations in UBA1 and Severe Adult-Onset Autoinflammatory Disease - DocWire News - October 30th, 2020
- JadiCell Stem Cells Licensed by Therapeutic Solutions International for Brain Injury Significantly Increased Survival of COVID-19 Patients in Double... - October 29th, 2020
- Impact Of Covid 19 On Adult Stem Cells Market 2020 Industry Challenges, Business Overview And Forecast Research Study 2026 - PRnews Leader - October 29th, 2020
- APstem Therapeutics Announces Successful FDA INTERACT Meeting Regarding AP-Skin-01, an Off-the-Shelf Allogeneic Stem Cell Product for the Treatment of... - October 29th, 2020
- Why Small Businesses Matter in Wilton (Reimagined): Interventional Orthopedics Connecticut - HamletHub - October 29th, 2020
- Covid-19 Impact On Orthopedic Regenerative Medicine Market 2020 Future Development, Manufacturers, Trends, Share, Size And Forecast to 2027 |... - October 29th, 2020
- Cell Expansion Market a comprehensive study by key players- Thermo Fisher Scientific, Inc, Becton, Dickinson and Company, Terumo BCT, Merck KGaA and... - October 24th, 2020
- Genmab Announces IFM, HOVON and Janssen Achieve Positive Topline Results in Second Part of Phase 3 CASSIOPEIA Study of Daratumumab in Multiple Myeloma... - October 24th, 2020
- What Is the Role of Allogeneic-SCT in Adult Acute Lymphoblastic Leukemia in the Era of Targeted Therapies? - Targeted Oncology - October 21st, 2020
- Cell Therapy Market Size, Share, Market Research and Industry Forecast Report, 2020-2027 (Includes Business Impact of COVID-19) - Eurowire - October 17th, 2020
- Global Stem Cell Therapy Market (Covid-19 updated report), Trends, Top Manufacturers, Exponential Growth and Forecast 2020 to 2027 By Reportspedia -... - October 17th, 2020