Delivering larger genes in gene therapy
Gene therapy has captured the imagination of scientists and science fiction fans alike. But as its development gains momentum, scientists...
Genetics and Gene Therapy Research
Bacteria’s Floating Genes Leave Scientists Baffled
Columbia researchers discovered that bacteria can create free-floating, temporary genes outside their chromosomes, challenging the long-held belief that all genetic...
Bacteria’s Floating Genes Leave Scientists Baffled
Columbia researchers discovered that bacteria can create free-floating, temporary genes outside their chromosomes, challenging the long-held belief that all genetic...
Machine learning revolutionizes gene delivery
Gene therapy could potentially cure genetic diseases but it remains a challenge to package and deliver new genes to specific...
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...
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)...
