Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Electroretinographic analyses of Rpe65-mutant rd12 mice: developing an in vivo bioassay for human gene therapy trials of Leber congenital amaurosis.

Molecular vision - 18 Sept 2007

Roman Alejandro J, Boye Sanford L, Aleman Tomas S, Pang Ji-jing, McDowell J Hugh, Boye Shannon E, Cideciyan Artur V, Jacobson Samuel G, Hauswirth William W

Abstract excerpt

PURPOSE: Dramatic restoration of retinal function has followed subretinal viral-mediated gene therapy in RPE65-deficient animal models of human Leber congenital amaurosis (LCA) caused by RPE65 mutations. Progress in early-phase clinical trials of RPE65-LCA prompted us to begin development of an in vivo bioassay of clinical grade vector stability for later-phase trials. METHODS: Naturally-occurring Rpe65-mutant...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Biological Assay
Share to Blindness

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to cis-trans-Isomerases

Share to Clinical Trials as Topic

Share to Electroretinography

Share to Optic Atrophy, Hereditary, Leber

Share to Photic Stimulation

Share to Retinal Degeneration

Source

Identifiers

PMID: 17960108

Genes

ERG
RPE65

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Electroretinographic analyses of Rpe65-mutant rd12 mice: developing an in vivo bioassay for human gene therapy trials of Leber congenital amaurosis.PMID 17960108Read ↗

Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.Semantic proximity Read ↗

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.Semantic proximity Read ↗

Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood–Onset Retinal DystrophySemantic proximity Read ↗

Retinal disease in Rpe65-deficient mice: comparison to human leber congenital amaurosis due to RPE65 mutations.Semantic proximity Read ↗

Long-term effect of gene therapy on Leber's congenital amaurosis.Semantic proximity Read ↗

Gene therapy for Leber congenital amaurosis: advances and future directions.Semantic proximity Read ↗

Clinical gene therapy for the treatment of RPE65-associated Leber congenital amaurosisSemantic proximity Read ↗

Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis.Semantic proximity Read ↗

Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.Semantic proximity Read ↗

Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy.Semantic proximity Read ↗

Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical EvidenceSemantic proximity Read ↗

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector AdministrationSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.