First patient dosed in the pediatric cohort of the Phase 1/2 trial of OPGx-LCA5; initial data on the cohort anticipated by Q3 2025New 12-month ...

Day 90 biopsy data reported from first 3 participants dosed in Phase 1/2 INSPIRE DUCHENNE trial —— Average microdystrophin expression of 110% ...

Organoids were cultured from healthy donor iPSC sources and differentiated using the human iPSC-derived Cerebral Organoid ...

Accurate monitoring of particle size and growth is essential to ensure scalability in the development of transfection vectors ...

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

Researchers review the ever-growing list of human viruses, examining their diversity, transmission pathways, and the urgent ...