The Vision Center at Children’s Hospital Los Angeles is developing a protocol for a phase 1 clinical trial for the first gene ...

May 2, 2007— -- A top eye scientist at the National Institutes of Health has called a new procedure that uses gene therapy to correct a condition that leads to blindness "an exciting ...

More information: Kvanta, A., et al. Interim safety and efficacy of gene therapy for RLBP1-associated retinal dystrophy: a phase 1/2 trial, Nature Communications (2024). DOI: 10.1038/s41467-024 ...

(CNN) — For her entire life, college student Olivia Cook had only a small degree of central vision. It was as if she was watching the world through a straw hole, and in dimly lit places, she ...

The most common form of color blindness is red-green color blindness. This happens when people are born without the type of ...

Four children born with a rare genetic form of severe blindness have all experienced significant improvements in their eyesight after being treated with an experimental new gene therapy. "We have ...

A child participant locates a small white object against a dark background after receiving gene therapy for LCA4. Credit: Michaelides et al, The Lancet/DOI 10.1016/S0140-6736(24)02812-5 ...

At least one gene therapy sold overseas has already crossed the $1-million price threshold, a treatment for a rare protein disorder launched in Europe. Manufacturer uniQure stopped selling the therapy ...

Interim safety and efficacy of gene therapy for RLBP1-associated retinal dystrophy: a phase 1/2 trial. Nature Communications , 2024; 15 (1) DOI: 10.1038/s41467-024-51575-4 Cite This Page : ...

In a phase 1/2 trial, researchers tested ATSN-101, a gene therapy for Leber congenital amaurosis 1, a rare genetic disorder that typically causes blindness in early childhood.

Scientists in the UK have successfully used gene therapy to restore some vision to legally blind children with an inherited retinal condition. All 11 children in the clinical trial saw ...

Q&A: Atsena Therapeutics’ gene therapy for inherited blindness shows promise. In 10 days after the gene therapy injection, on average, there was a 100-fold improvement in vision.