Ontology highlight
ABSTRACT:
SUBMITTER: Banin E
PROVIDER: S-EPMC4817879 | biostudies-literature | 2015 Sep
REPOSITORIES: biostudies-literature
Banin Eyal E Gootwine Elisha E Obolensky Alexey A Ezra-Elia Raaya R Ejzenberg Ayala A Zelinger Lina L Honig Hen H Rosov Alexander A Yamin Esther E Sharon Dror D Averbukh Edward E Hauswirth William W WW Ofri Ron R
Molecular therapy : the journal of the American Society of Gene Therapy 20150619 9
Achromatopsia is a hereditary form of day blindness caused by cone photoreceptor dysfunction. Affected patients suffer from congenital color blindness, photosensitivity, and low visual acuity. Mutations in the CNGA3 gene are a major cause of achromatopsia, and a sheep model of this disease was recently characterized by our group. Here, we report that unilateral subretinal delivery of an adeno-associated virus serotype 5 (AAV5) vector carrying either the mouse or the human intact CNGA3 gene under ...[more]