Gene Therapy Restores Vision – Neuroscience News
Summary: A new gene therapy has restored vision in patients with Leber congenital amaurosis type I (LCA1), a rare genetic...
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Gene therapy gets a turbo boost from researchers
Graphical abstract. Credit: Nucleic Acids Research (2024). DOI: 10.1093/nar/gkae534 For decades, scientists have dreamed of a future where genetic diseases,...
Streamlining Viral Gene Therapy Development
While viral vector-based gene therapies, such as those employing adeno-associated viruses, are increasing in popularity, scientists face several challenges when...
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...
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...
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)...
