Share this Article
You are free to share this article under the Attribution 4.0 International license.
A new gene therapy turns glial cellsabundant support cells in the braininto neurons, repairing damage that results from stroke and significantly improving motor function in mice.
Once researchers further develop the NeuroD1-based gene therapy, it could potentially help to treat stroke, which is a leading cause of disability in the US, with 800,000 new stroke patients every year.
The current treatment for stroke has a narrow time window, typically within a few hours after the occurrence of stroke, says lead author Yuchen Chen, a postdoctoral fellow at Penn State. Many patients cannot receive the treatment in time and as a result, often suffer from permanent disability caused by irreversible neuronal loss. There is an urgent need to develop a new therapy to regenerate new neurons and restore lost brain functions among stroke patients.
The human brain has approximately 86 billion neurons. While mini-strokes can be tolerated, moderate stroke involving the loss of billions of neurons leaves detrimental effects that do not spontaneously recover.
So, the critical question that is still unanswered in the neuroregeneration field is how can we regenerate billions of new neurons in a patients brain after stroke? says Gong Chen, professor of biology and chair in life sciences. The biggest obstacle for brain repair is that neurons cannot regenerate themselves. Many clinical trials for stroke have failed over the past several decades, largely because none of them can regenerate enough new neurons to replenish the lost neurons.
The reserchers pioneered a new approach to regenerate functional neurons using glial cells, a group of cells surrounding every single neuron in the brain that provide essential support to neurons. Unlike neurons, glial cells can divide and regenerate themselves, especially after brain injury.
I believe that turning glial cells that are already present in the brain into new neurons is the best way to replenish the lost neurons, says Gong Chen. These glial cells are the neighbors of the dead neurons in the brain and are likely to share the same ancestral cellular lineage.
The team previously reported that a single genetic neural factor, NeuroD1, could directly convert glial cells into functional neurons inside mouse brains with Alzheimers disease, but the total number of neurons generated was limited. The research team believed that this limited regeneration was due to the retroviral system used to deliver NeuroD1 to the brain.
In the current study, the research team used the AAV viral system, which is now the first choice for gene therapy in the nervous system, to deliver NeuroD1 into mouse motor cortex that a stroke had damaged.
Many neurons die after stroke but surviving glial cells can proliferate and form a glial scar in the stroke areas. The researchers designed their AAV system to express NeuroD1 preferentially in the glial cells that form these scars, turning them directly into neuronal cells. Such direct glia-to-neuron conversion technology not only increased neuronal density in the stroke areas, but also significantly reduced brain tissue loss the stroke caused.
Interestingly, the newly converted neurons showed similar neuronal properties to the neurons that were lost after stroke. This suggests a potential impact of the local glial lineage on the converted neuronal identity.
The most exciting finding of this study is to see the newly converted neurons being fully functional in firing repetitive action potentials and forming synaptic networks with other preexisting neurons, says Gong Chen. They also send out long-range axonal projections to the right targets and facilitate motor functional recovery.
A separate collaborative work led by Gregory Quirk, a professor at the University of Puerto Rico, further tested the NeuroD1-based gene therapy in a rat stroke model. Quirk and colleagues also found that this direct glia-to-neuron conversion technology can rescue cognitive functional deficits stroke induces.
Because glial cells are everywhere in the brain and can divide to regenerate themselves, our study provides the proof-of-concept that glial cells in the brain can be tapped as a fountain of youth to regenerate functional new neurons for brain repair not only for stroke but also for many other neurological disorders that result in neuronal loss, says Yuchen Chen. Our next step is to further test this technology and ultimately to translate it into clinically effective therapies to benefit millions of patients worldwide.
A paper describing the new therapy appears in the journal Molecular Therapy.
Additional researchers at Penn State and the University of Puerto Rico contributed to the work. The US National Institutes of Health and the Penn State Charles H. Skip Smith Endowment Fund supported the research.
Source: Penn State
Read more from the original source:
- Gyrolab AAVX Titer Kit Introduced to Support Cell and Gene Therapy Market - Technology Networks - September 2nd, 2020
- Taysha Gene Therapies Builds Experienced Executive Leadership Team to Advance Pipeline of Gene Therapies for Monogenic CNS Disease in Both Rare and... - September 2nd, 2020
- First Gene Therapy Products Approved in Brazil - Lexology - September 2nd, 2020
- Gene Therapy Market is projected to be worth USD 10 Billion by 2030, growing at an annualized rate of over 40% - The News Brok - September 2nd, 2020
- Prevail Therapeutics to Present at Upcoming Investor Conferences - Yahoo Finance - September 2nd, 2020
- Orchard Therapeutics Announces Additional Interim Results from Proof-of-Concept Study of OTL-203 for MPS-I - BioSpace - September 2nd, 2020
- The Safety and Clinical Activity of Gene-engineered T-cell Therapy Targeting HPV-16 E7 in Epithelial Cancers - Pharmacy Times - September 2nd, 2020
- AGTC to Host Management Update on September 9 at 8:00 am ET to Discuss Planned Phase 2/3 XLRP Trial, Share Additional XLRP Data and Report Fourth... - September 2nd, 2020
- Qualitative Analysis and Competitive Industry, CCOVID-19 Scenario of Personalized Gene Therapy Treatment Market - The News Brok - September 2nd, 2020
- Sorrento Announces the Closing of Its Acquisition of SmartPharm to Build Next Generation G-MAB-Encoded Plasmid DNA For Cost-Efficient and In Vivo... - September 2nd, 2020
- In the face of COVID-19, cell and gene therapy space shows 'remarkable resilience:' report - FierceBiotech - August 10th, 2020
- Gene Therapy Promising in BCG-Unresponsive Bladder Cancer - Medscape - August 10th, 2020
- Evolution and Expansion of Therapies in the Global Cell and Gene Therapy Tools and Reagents Market 2020-2024 - PRNewswire - August 10th, 2020
- How Gene Therapy Helped Conner Run : Short Wave - NPR - August 10th, 2020
- Preparing for an influx of cell and gene therapy approvals - - pharmaphorum - August 10th, 2020
- Catalent's Harmans Site Approved To Manufacture AveXis' Gene Therapy - Contract Pharma - August 10th, 2020
- Sangamo and Novartis partner on gene therapies for autism - BioPharma-Reporter.com - August 10th, 2020
- Global Gene Therapy in Oncology Market 2020 Growth Statistics, New Opportunities, Competitive Outloo - PharmiWeb.com - August 10th, 2020
- Research shows promising results for LHON gene therapy - Ophthalmology Times - August 10th, 2020
- The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.90% during the period 20192025 - Yahoo Finance - August 10th, 2020
- Detailed Information on Gene Therapy Market 2020 | Covid-19 Impact Analysis | Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio,... - August 10th, 2020
- Global Cell & Gene Therapy Market Outlook and Forecast 2020-2025 - GlobeNewswire - August 9th, 2020
- Coronavirus Impact Editon of Gene Therapy for Mucopolysaccharidosis Market Research Study 2020 Future Development, Top Manufacturers, Technological... - August 9th, 2020
- The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.90% during the period 20192025 - Press Release - Digital... - August 9th, 2020
- Orgenesis Second Quarter 2020 Revenue Increases 55% Reflecting Progress of POCare Platform - GlobeNewswire - August 9th, 2020
- Regulatory Focus, July issue: Cell and gene therapy - Regulatory Focus - August 8th, 2020
- G-CON PODs Selected for Expression Therapeutics' Gene Therapy Manufacturing Facility - PR Web - August 8th, 2020
- CRISPR co-discoverer on the gene editor's pandemic push - Axios - August 8th, 2020
- Voyager Therapeutics Provides Update on AbbVie Vectorized Antibody Collaborations - GlobeNewswire - August 8th, 2020
- Gene therapy - Wikipedia - August 8th, 2020
- Gene therapy - Mayo Clinic - August 8th, 2020
- gene therapy | Description, Uses, Examples, & Safety ... - August 8th, 2020
- What Is Gene Therapy? How Does It Work? | FDA - August 8th, 2020
- 112M Fund Launched to Commercialize UCLs Gene Therapy... - Labiotech.eu - August 8th, 2020
- UK gene therapy pioneer tilts at $125m NASDAQ IPO - Business Weekly - August 7th, 2020
- Of mice and memories: Gene therapy study returns function in Alzheimer's mice - ABC News - August 7th, 2020
- Tessa Therapeutics Announces Results from Two Independent Phase 1/2 Trials of Autologous CD30 CAR-T Cell Therapy in Patients with Relapsed or... - August 7th, 2020
- Cancer Gene Therapy Market Latest Treatment Methodology 2020 to 2025 - Owned - August 7th, 2020
- Kriya Therapeutics announces the establishment of its internal manufacturing facility for process development and scalable cGMP production of gene... - August 7th, 2020
- Regenxbio announces positive one-year data on wet AMD gene therapy - Seeking Alpha - August 7th, 2020
- Covid-19 Forces Gene Therapy Companies to Shift... - Labiotech.eu - August 5th, 2020
- Cell and gene therapies: For biotechs, collaboration is key to successful innovation - BioPharma Dive - August 5th, 2020
- The Time for DMD Gene Therapy is Now: A Chat with the MDA - BioSpace - August 5th, 2020
- Gene Therapy Beats the Blood-Brain Barrier To Cure Cat Disease - Technology Networks - August 5th, 2020
- Analysis of COVID-19 Gene Therapy Market - Market Research Posts - August 5th, 2020
- World-first gene therapy reverses Alzheimer's memory loss in mice - New Atlas - August 5th, 2020
- Gene Therapy Market is projected to grow at an annualized rate of 45%, till 2030 - Market Research Posts - August 5th, 2020
- Spark Therapeutics Recognized for Fourth-straight Year as One of Philadelphia's Best Places to Work by the Philadelphia Business Journal - BioSpace - August 5th, 2020
- AGTC to Present at the Wedbush PacGrow Virtual Healthcare Conference on August 11, 2020 - BioSpace - August 5th, 2020
- Global Hemophilia Gene Therapy Market is estimated to Experience a Notable Rise in the coming era by Spark Therapeutics, Ultragenyx, Shire PLC,... - August 5th, 2020
- MEDIPAL HOLDINGS Partners with CRYOPORT to Bring Temperature-Controlled Supply Chain Solutions to the Japanese Market - PRNewswire - August 5th, 2020
- Can progenitor cells go further than gene therapy in retinal disorders? A biotech's answer shines in PhIIb - Endpoints News - August 4th, 2020
- Oxford Biomedica Signs Three Year Clinical Supply Agreement with Axovant Gene Therapies for Manufacture and Supply of AXO-Lenti-PD - GlobeNewswire - August 4th, 2020
- Cell and Gene Therapy Market 2020 | Enormous Growth with Recent Trends & Demand By Top Vendors JW CreaGene, Vericel, Tego Sciences, CHIESI... - August 4th, 2020
- Comprehensive Report on Gene Therapy for Ovarian Cancer Market 2020 | Trends, Growth Demand, Opportunities & Forecast To 2026 | Takara Bio, VBL... - August 4th, 2020
- Gene therapy reverses memory loss from Alzheimer's in mice - BioNews - August 3rd, 2020
- Hemophilia Gene Therapy Industry Market Analysis by Size, Share, Growth, Application, Segmentation and Forecast to 2025 - Express Journal - August 3rd, 2020
- Dyne Therapeutics Appoints Susanna High as Chief Operating Officer - Business Wire - August 3rd, 2020
- Cell and Gene Therapy Industry Market with manufacturers, Application, regions and SWOT Analysis 2025 - CueReport - August 3rd, 2020
- Investing in Advanced Manufacturing to Support Public Health - FDA.gov - August 3rd, 2020
- Genetic Studies Could Pave the Way to New Pain Treatments - Pain News Network - August 3rd, 2020
- Oxford Biomedica Signs Development, Manufacture & License Agreement with Beam Therapeutics Inc., for LentiVector Platform for Next Generation... - August 3rd, 2020
- AZ partners with Daiichi on ADC therapy - BioPharma-Reporter.com - August 3rd, 2020
- Getting gene therapy to the brain | Penn Today - Penn Today - August 2nd, 2020
- Ocugen Granted FDA Orphan Drug Designation for OCU400 (AAV-hNR2E3) Gene Therapy for the Treatment of RHO Mutation-Associated Retinal Degenerative... - August 2nd, 2020
- Axovant Signs Three-Year Clinical Supply Agreement With Oxford Biomedica for Manufacturing and Supply of AXO-Lenti-PD - BioSpace - August 2nd, 2020
- Gene Therapy for Inherited Genetic Disorders Market size in terms of volume and value 2019-2025 - Market Research Correspondent - August 2nd, 2020
- Gene Therapy Market Professional Industry Overview 2020 Segmentation and Development Analysis BY 2027 - Science Examiner - August 2nd, 2020
- Greenwich swimmers take the plunge to fight cancer - Greenwich Time - August 2nd, 2020
- Cell and Gene Therapy Market Overview, Growth & Advancement to (2020-2025) - Owned - August 2nd, 2020
- Movers & Shakers, July 31 | BioSpace - BioSpace - August 2nd, 2020
- REGENXBIO To Host Conference Call on August 6 to Discuss Second Quarter 2020 Financial Results and Recent Operational Highlights - Herald-Mail Media - August 2nd, 2020
- Catharpin mom helps raise national awareness for her young sons disease - WDVM 25 - August 2nd, 2020
- Spinraza-Zolgensma Combination Well-tolerated in Children with SMA... - SMA News Today - August 2nd, 2020
- Vineti and EVERSANA form strategic alliance to advance patient-centered services and supply chain automation for regenerative medicine - PRNewswire - August 2nd, 2020
- Orchard Therapeutics to Webcast Conference Call of Second Quarter 2020 Financial Results - GlobeNewswire - August 2nd, 2020
- Expression Therapeutics Announces Bill Swaney as VP of Manufacturing and the Construction of Cell and Gene Therapy Manufacturing - Yahoo Finance - August 2nd, 2020
- REGENXBIO To Host Conference Call on August 6 to Discuss Second Quarter 2020 Financial Results and Recent Operational Highlights - PRNewswire - August 2nd, 2020
- Global Gene Therapy Market Market Analysis by SWOT, Investment, Future Growth and Major Key Players 2020 to 2025 - Research Newspaper - August 2nd, 2020
- Cell and Gene Therapy Tools, and Reagents: Global Markets - PRNewswire - August 1st, 2020