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

Gene therapy: light is finally in the tunnel.

Protein & cell - 1 Dec 2011

Cao Huibi, Molday Robert S, Hu Jim

Abstract excerpt

After two decades of ups and downs, gene therapy has recently achieved a milestone in treating patients with Leber's congenital amaurosis (LCA). LCA is a group of inherited blinding diseases with retinal degeneration and severe vision loss in early infancy. Mutations in several genes, including RPE65, cause the disease. Using adeno-associated virus as a vector, three independent teams of investigators have...

Read the complete abstract on PubMed

Topics

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

Share to Adaptive Immunity
Share to cis-trans-Isomerases
Share to Dependovirus

Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Share to Gene Targeting

Share to Leber Congenital Amaurosis

Share to Liposomes

Share to Retina

Share to Retroviridae

Source

Open in PubMed Open full text

Identifiers

PMID: 22231356
DOI: 10.1007/s13238-011-1126-y
PMCID: PMC4728158

Genes

RPE65

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Gene therapy: light is finally in the tunnel.PMID 22231356Read ↗

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

Retinal gene therapySemantic proximity Read ↗

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

[Gene therapy for vision restoration in patients with Leber congenital amaurosis (LCA) due to RPE65 gene mutations: beginning the phase IV trial].Semantic proximity Read ↗

The Status of RPE65 Gene Therapy Trials: Safety and Efficacy.Semantic proximity Read ↗

Gene therapy for eye as regenerative medicine? Lessons from RPE65 gene therapy for Leber's Congenital Amaurosis.Semantic proximity Read ↗

Non-viral gene therapy for Leber’s congenital amaurosis: progress and possibilitiesSemantic proximity Read ↗

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

Voretigene Neparvovec and Gene Therapy for Leber’s Congenital Amaurosis: Review of Evidence to DateSemantic proximity Read ↗

Safety and efficacy of gene transfer for Leber's congenital amaurosis.Semantic proximity Read ↗

The Role of Gene Therapy in the Treatment of Retinal Diseases: A ReviewSemantic proximity Read ↗

Gene Therapy of Inherited Retinopathies: A Long and Successful Road from Viral Vectors to PatientsSemantic proximity Read ↗

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