Clinical Trial
Single-dose gene therapy restores hearing in children born deaf
Eleven children with a rare inherited deafness regained significant hearing after a one-time inner-ear gene therapy, sustained at one year.
Clinical Trial
Peer-reviewed
Complete human genome finally sequenced — including the missing 8%
The gapless T2T reference fills in centromeres and repeats long thought unreadable, opening new frontiers in genetics.
Clinical Trial
CRISPR edit reverses high cholesterol in first-in-human trial
A one-time base edit lowered LDL cholesterol by 55% in patients, a proof of concept for in-vivo gene editing of…