Insulin injections cancontrol diabetes, but patients still experience serious complications such as kidney disease and skin infections. Transplanting pancreatic tissues containing functional insulin-producing beta cells is of limited use, because donors are scarce and patients must take immunosuppressant drugs afterward.
Now, scientists atWashington University in St. Louis havedeveloped a way to use gene editing system CRISPR-Cas9 to edit a mutation in human-induced pluripotent stem cells (iPSCs) and then turnthem into beta cells. When transplanted into mice, the cells reversed preexisting diabetes in a lasting way, according to results published in the journal Science Translational Medicine.
While the researchers used cells from patients with Wolfram syndromea rare childhood diabetes caused by mutations in the WFS1 genethey argue that the combination of a gene therapy with stem cells could potentially treat other forms of diabetes as well.
Virtual Clinical Trials Online
This virtual event will bring together industry experts to discuss the increasing pace of pharmaceutical innovation, the need to maintain data quality and integrity as new technologies are implemented and understand regulatory challenges to ensure compliance.
One of the biggest challenges we faced was differentiating our patient cells into beta cells. Previous approaches do not allow for this robust differentiation. We use our new differentiation protocol targeting different development and signaling pathways to generate our cells, the studys lead author, Kristina Maxwell, explained in a video statement.
Making pancreatic beta cells from patient-derived stem cells requires precise activation and repression of specific pathways, and atthe right times, to guide the development process. In a recent Nature Biotechnology study, the team described a successful method that leverages the link between a complex known as actin cytoskeleton and the expression of transcription factors that drive pancreatic cell differentiation.
This time, the researchers applied the technology to iPSCs from two patients with Wolfram syndrome. They used CRISPR to correct the mutated WFS1 gene in the cells and differentiated the edited iPSCs into fully functional beta cells.
After transplanting the corrected beta cells into diabetic mice, the animals saw their blood glucose drop quickly, suggesting the disease had been reversed. The effect lasted for the entire six-month observation period, the scientists reported. By comparison, those receiving unedited cells from patients were unable to achieve glycemic control.
RELATED:CRISPR Therapeutics, ViaCyte team up on gene-edited diabetes treatment
The idea of editing stem cells with CRISPR has already attracted interest in the biopharma industry. Back in 2018, CRISPR Therapeutics penned a deal with ViaCyte to develop off-the-shelf, gene-editing stem cell therapies for diabetes. Rather than editing iPSCs from particular patients themselves to correct a faulty gene, the pairs lead project used CRISPR to edit healthy cells so that they lackedthe B2M gene and expressed PD-L1 to protect against immune attack. The two companies unveiled positive preclinical data inSeptember.
Other research groups working on gene therapy or stem cells for diabetes include a Harvard University scientist and his startup Semma Therapeutics, whichdeveloped a method for selecting beta cells out of a mixture of cells developed from PSCs. Scientists at the University of Wisconsin-Madison recently proposed that removing the IRE1-alpha gene in beta cells could prevent immune T cells from attacking them in mice with Type 1 diabetes.
The Washington University team hopes its technology may help Type 1 diabetes patients whose disease is caused by multiple genetic and environmental factors as well as the Type 2 form linked to obesity and insulin resistance.
We can generate a virtually unlimited number of beta cells from patients with diabetes to test and discover new drugs to hopefully stop or even reverse this disease, Jeffrey Millman, the studys co-senior author, said in the video statement. Perhaps most importantly, this technology now allows for the potential use of gene therapy in combination with the patients own cells to treat their own diabetes by transplantation of lab-grown beta cells.
- 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
- Oxford Biomedica Signs Three Year Clinical Supply Agreement with Axovant Gene Therapies for Manufacture and Supply of AXO-Lenti-PD - PharmiWeb.com - July 31st, 2020
- Expression Therapeutics Announces Bill Swaney as VP of Manufacturing and the Construction of Cell and Gene Therapy Manufacturing - BioSpace - July 31st, 2020
- CPI, the Cell and Gene Therapy Catapult and MicrofluidX announce development of technology to rapidly scale up cell and gene therapy manufacture -... - July 31st, 2020
- Navigating Complexity in Oncology Cell and Gene Therapy Clinical Trials - Clinical OMICs News - July 31st, 2020
- Orchard Therapeutics to Webcast Conference Call of Second Quarter 2020 Financial Results - BioSpace - July 31st, 2020
- Genprex CEO Participates in a Second-Round Live Interview on the Big Biz Show - Business Wire - July 31st, 2020
- Ongoing Trial Assesses New Anti-VEGF Gene Therapy Injection for nAMD - MD Magazine - July 30th, 2020
- Increasing Government Investments and Favorable Policies to Aid the Growth of the Cancer Gene Therapy Market2020 - Owned - July 30th, 2020
- Cell and Gene Therapy Tools, and Reagents: Global Markets - cnweekly - July 30th, 2020
- Consumer Demand for Eco-friendly Products Set to Boost the Prospects of the Cancer Gene Therapy Market2020 - Owned - July 30th, 2020