Intravitreal injection of AAV7m8.hLCA5 restores photoreceptor function in Lca5-/- mice to nearly WT levels.

Puya Aravand(Penn Presbyterian Medical Center), Sergei Nikonov(Penn Presbyterian Medical Center), Arkady Lyubarsky(Penn Presbyterian Medical Center), Jieyan Pan(Penn Presbyterian Medical Center), Jean Bennett(Penn Presbyterian Medical Center), Ji Yun Song(Penn Presbyterian Medical Center), Shangzhen Zhou(Penn Presbyterian Medical Center)
Investigative Ophthalmology & Visual Science
June 23, 2017
Cited by 0

Related Papers

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
|New England Journal of Medicine|2008|2.1k
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
|The Lancet|2009|864
Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration
|Molecular Therapy|2009|551
Mutation of a nuclear receptor gene, NR2E3, causes enhanced S cone syndrome, a disorder of retinal cell fate
|Nature Genetics|2000|506
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial
|The Lancet|2016|464