A team of doctors and scientists have successfully treated a rare genetic condition with the first-ever personalized ...

Ask scientists which gene-editing tool is most needed to advance gene therapy, and they'd probably describe a system that's ...

Ask scientists what gene editing tool is most needed to advance gene therapy, and they'd probably describe a system that's now close to realization in the labs of Samuel Sternberg at Columbia ...

Biopharmas have been scaling back on AAV research for sometime now. Find out why and what's on the horizon for drug delivery ...

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The technique used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other ...

When we published our 2024 report, children suffering from Leber Congenital Amaurosis 4 (LCA4)—a severe inherited retinal ...

A new gene editor takes advantage of CRISPR-associated proteins to insert whole genes into the genome, scientists report.

In this op-ed, Charles Gersbach explores how genetic tuning is emerging as a next-generation approach to modulating gene ...

Boehringer Ingelheim (BI) and the UK Cystic Fibrosis Gene Therapy Consortium announced a global collaboration to develop a first-in-class, long-term therapy for patients with cystic fibrosis (CF).

A range of other rare inherited diseases are now likely to be curable by similar procedures, the research team said ...