Long-term effects of retinal gene therapy in childhood blindness
AF Wright - New England Journal of Medicine, 2015 - Mass Medical Soc
AF Wright
New England Journal of Medicine, 2015•Mass Medical SocCurrent ocular imaging techniques provide extraordinarily high-resolution visualization of
the retinal layers, amounting to what has been called in vivo microscopy. Combine this with
a battery of sophisticated retinal-function tests, and the retina becomes unusually accessible
in the evaluation of trials of retinal gene therapy for the treatment of conditions such as
Leber's congenital amaurosis. Leber's congenital amaurosis is a group of inherited retinal
dystrophies associated with blindness early in life and can be caused by mutations in at …
the retinal layers, amounting to what has been called in vivo microscopy. Combine this with
a battery of sophisticated retinal-function tests, and the retina becomes unusually accessible
in the evaluation of trials of retinal gene therapy for the treatment of conditions such as
Leber's congenital amaurosis. Leber's congenital amaurosis is a group of inherited retinal
dystrophies associated with blindness early in life and can be caused by mutations in at …
Current ocular imaging techniques provide extraordinarily high-resolution visualization of the retinal layers, amounting to what has been called in vivo microscopy. Combine this with a battery of sophisticated retinal-function tests, and the retina becomes unusually accessible in the evaluation of trials of retinal gene therapy for the treatment of conditions such as Leber’s congenital amaurosis.
Leber’s congenital amaurosis is a group of inherited retinal dystrophies associated with blindness early in life and can be caused by mutations in at least 19 different genes. Loss-of-function mutations in one of these genes, RPE65, account for 5 to 10% of recessive Leber’s . . .
The New England Journal Of Medicine