Reposted by Huma R
Genetic therapy gives infants life-changing improvements in sight. #TheLancet
"Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study"
www.thelancet.com/journals/lan...
thelancet.com
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Our findings indicate that young children with AIPL1-related retinal dystrophy benefited
substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual
acuity and functional vi...