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Science Daily Gene Therapy News
ScienceDaily is best known for showcasing the top science news stories from the world’s leading universities and research organizations. These current stories on gene therapy are selected from among dozens of press releases and other materials submitted to ScienceDaily every day.
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- Updates on gene therapy for ‘bubble boy’ disease and cellular immunotherapy at ASGCT
- AAV Durability: The Road to a One-Shot Cure
- Gene Therapy Sector Needs Better Cell Lines for Vector Production
- Gene Therapy Turns to the Use of Synthetic Biology Principles
- Lentiviral vector-based gene therapy demonstrates long-term safety and efficacy for Wiskott-Aldrich Syndrome
- Nanoscope Therapeutics announces FDA clearance of IND for MCO-010 gene therapy in Stargardt patients
- Avrobio stops work on rare disease gene therapy after unexpected study results
- New Bespoke Gene Therapy Consortium Is Welcome Development
- DMD Gene Therapy Candidate RGX-202 Earns Orphan Drug Status
- NIH and FDA Lead Coalition to Accelerate Gene Therapy Development for Rare Diseases
- FDA: Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations
- Fourth Boy Dies in Trial of Astellas Gene Therapy Candidate
- New gene-delivering workhorse could make gene therapy safer, more effective for muscle diseases
- Lysogene Announces First Patient in the United States Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis
- FDA allows Novartis gene therapy trials to resume after nearly 2 year pause
- Gene therapy delivered to the brain shows promise in children with rare neurodegenerative disease
- A year after getting UniQure's gene therapy, hemophilia patients are still doing better
- Decades after a tragic failure, gene therapy successfully treats a rare liver disease
- New gene therapy restores dystrophin protein in patients with Duchenne muscular dystrophy
- New approach to CAR T gene therapy yields efficient and longer-lasting response against HIV
- EMA Launches Safety Review After Gene Therapy Trial Patient Develops Blood Cancer
- Gene therapy trials for sickle cell disease halted after two patients develop cancer
- Manufacturing Considerations for Licensed and Investigational Cellular and Gene Therapy Products During COVID-19 Public Health Emergency
- Liver tumor in gene therapy recipient raises concerns about virus widely used in treatment
- Gene Therapy in One Eye Improves Vision in Both Eyes
- NIH project aims to maximize efficiency of AAV platform for gene therapy
- COVID-19, manufacturing challenges limit cell and gene therapy progress, FDA official says
- Gene Therapy in Mesothelioma Patients
- FDA grants orphan drug designation for LHON gene therapy
- New HIV Gene Therapy, CAR-T Treatments Could be on the Horizon for Patients