Year: 2008
Type: article
Abstract: Two new studies in the New England Journal of Medicine report improvements in the vision of patients with a genetic degenerative disease of vision subsequent to injection of adeno-associated virus (AAV) into the subretinal space.1,2 Both studies targeted Leber's congenital amaurosis, a group of inherited diseases causing reduced vision early in life and progressive blindness. The form of the disease treated in these studies is caused by mutation of the retinal pigment epithelium–specific 65-kDa protein gene (RPE65).
Source: Molecular Therapy
DOI: 10.1038/mt.2008.123
Author: David A Williams
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $4,500
OpenAlex ID: W2084628361