Research suggests gene therapy may help improve vision in patients with Leber’s congenital amaurosistomet

Following a HealthDay story, Medscape (8/20) reported that, according to a letter published Aug. 13 in the New England Journal of Medicine, “three patients with Leber’s congenital amaurosis (LCA) demonstrated improved vision after injection of a viral vector carrying the wild type of the impaired gene.” For the study, researchers from the University of Pennsylvania worked with “three LCA patients in their twenties,” delivering by rAAV2 viral vector to the retina “normal copies of RPE65,” a gene that “normally codes for a protein required for a retina-specific version of vitamin A needed for photoreceptor cell function.” Shortly after treatment, all three legally blind patients “demonstrated improved light sensitivity, but only in the area of the retina that received the injection. Within weeks after treatment,” however, “they could detect dim lights at a level not previously perceived.” Notably, one year after treatment, “the treated retinal region of one patient was more sensitive than the natural fovea and functioned as a pseudo-fovea.” For more inforamtion on eye and vision care visit


Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out /  Change )

Google+ photo

You are commenting using your Google+ account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )


Connecting to %s

%d bloggers like this: