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 rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa.

Proceedings of the National Academy of Sciences of the United States of America - 7 Feb 2012

Beltran William A, Cideciyan Artur V, Lewin Alfred S, Iwabe Simone, Khanna Hemant, Sumaroka Alexander, Chiodo Vince A, Fajardo Diego S, Román Alejandro J, Deng Wen-Tao, Swider Malgorzata, Alemán Tomas S, Boye Sanford L, Genini Sem, Swaroop Anand, Hauswirth William W, Jacobson Samuel G, Aguirre Gustavo D

Abstract excerpt

Hereditary retinal blindness is caused by mutations in genes expressed in photoreceptors or retinal pigment epithelium. Gene therapy in mouse and dog models of a primary retinal pigment epithelium disease has already been translated to human clinical trials with encouraging results. Treatment for...

Read the complete abstract on PubMed

Topics

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

Share to Blindness
Share to Eye Proteins

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Genetic Diseases, X-Linked

Share to Genetic Therapy

Share to Open Reading Frames

Share to Opsins

Share to Photoreceptor Cells, Vertebrate

Share to Retinitis Pigmentosa

Source

Open in PubMed Open full text

Identifiers

PMID: 22308428
DOI: 10.1073/pnas.1118847109
PMCID: PMC3277562

Genes

RPGR
GRK1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026. Prepared record publication-22308428-2026-09-04.2. Manifest publication-graph-cohort-2026-09-04.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 4 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa.PMID 22308428Read ↗

Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of diseaseSemantic proximity Read ↗

Gene augmentation for X-linked retinitis pigmentosa caused by mutations in RPGR.Semantic proximity Read ↗

Successful Gene Therapy in the RPGRIP1-deficient Dog: a Large Model of Cone–Rod DystrophySemantic proximity Read ↗

Patients and animal models of CNGβ1-deficient retinitis pigmentosa support gene augmentation approach.Semantic proximity Read ↗

Pre-Clinical and Clinical Advances in Gene Therapy of X-Linked Retinitis Pigmentosa: Hope on the HorizonSemantic proximity Read ↗

Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR.Semantic proximity Read ↗

RPGR-associated retinopathy: clinical features, molecular genetics, animal models and therapeutic options.Semantic proximity Read ↗

AAV-RPGR Gene Therapy Rescues Opsin Mislocalisation in a Human Retinal Organoid Model of RPGR-Associated X-Linked Retinitis Pigmentosa.Semantic proximity Read ↗

Gene Therapy in a Large Animal Model of PDE6A-Retinitis PigmentosaSemantic proximity Read ↗

Molecular Strategies for RPGR Gene TherapySemantic proximity Read ↗

Research Models and Gene Augmentation Therapy for CRB1 Retinal DystrophiesSemantic proximity Read ↗

Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa.Semantic proximity Read ↗

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