Vision 1 year after gene therapy for Leber's congenital amaurosis
AV Cideciyan, WW Hauswirth, TS Aleman… - … England Journal of …, 2009 - Mass Medical Soc
New England Journal of Medicine, 2009•Mass Medical Soc
To the Editor: Leber's congenital amaurosis, a common cause of blindness in infants and
children, 1 recently became the first human genetic retinal disease to show improved vision
in response to treatment. Patients with mutations in the gene encoding retinal pigment
epithelium–specific 65-kD protein (RPE65) had gains in vision within weeks after subretinal
injection of a vector containing the gene in one eye. 2–5 At 1-year follow-up after gene
therapy, the three young adult patients in our trial4, 5 remained without serious adverse …
children, 1 recently became the first human genetic retinal disease to show improved vision
in response to treatment. Patients with mutations in the gene encoding retinal pigment
epithelium–specific 65-kD protein (RPE65) had gains in vision within weeks after subretinal
injection of a vector containing the gene in one eye. 2–5 At 1-year follow-up after gene
therapy, the three young adult patients in our trial4, 5 remained without serious adverse …
To the Editor: Leber's congenital amaurosis, a common cause of blindness in infants and children,1 recently became the first human genetic retinal disease to show improved vision in response to treatment. Patients with mutations in the gene encoding retinal pigment epithelium–specific 65-kD protein (RPE65) had gains in vision within weeks after subretinal injection of a vector containing the gene in one eye.2–5 At 1-year follow-up after gene therapy, the three young adult patients in our trial4,5 remained without serious adverse events.
A noteworthy observation in one patient at 1 year after treatment prompted further studies. For . . .
The New England Journal Of Medicine