Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate
Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden’s rare, inherited immune deficiency. But it’s not...
Genetics and Gene Therapy Research
Baby born deaf can hear after breakthrough gene therapy
Opal Sandy from Oxfordshire is the first patient treated in a global gene therapy trial, which shows 'mind-blowing' results. She...
New gene therapy for muscular dystrophy offer
image: Muscular dystrophy gene therapy delivery system researchers Jeffrey Chamberlain (left) and Hichem Tasfaout in their laboratory at the University of...
Method that silences specific genes while leaving others unaffected could aid research and therapy
Fusion of translational repressors to dPspCas13b enhances translational repression efficacy. Credit: Nature Communications (2024). DOI: 10.1038/s41467-024-46412-7 RIKEN researchers have developed...
Gene therapy milestone at SickKids offers hope for children with SPG50 neurodegenerative disorder
When Michael Pirovolakis received an individualized gene therapy in a single-patient clinical trial at The Hospital for Sick Children (SickKids)...
Gene Therapy Market Surges to USD 50.6 Billion by 2031,
Lewes, Delaware, July 17, 2024 (GLOBE NEWSWIRE) -- The Global Gene Therapy Market Size is projected to grow at a...
CHARMed collaboration creates a potent therapy candidate for fatal prion diseases | MIT News
Drug development is typically slow: The pipeline from basic research discoveries that provide the basis for a new drug to...
Bibliometric analysis of global research trends in adeno-associated virus vector for gene therapy (1991-2022)
1 IntroductionGene therapy is a therapeutic approach aimed at introducing foreign genetic material into target cells using non-viral or viral...
Department of Health – Abu Dhabi and Opus Genetics Announce Collaboration to Advance Ground-Breaking Gene Therapy Research for Inherited Retinal Diseases
ABU DHABI, UAE, June 4, 2024 /PRNewswire/ -- During the BIO 2024 International Convention, the Department of Health – Abu...
