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

doi:10.1016/S0140-6736(09)61836-5

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

Topics Community

Back to search

Article

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

Lancet (London, England) - 7 Nov 2009

Maguire Albert M, High Katherine A, Auricchio Alberto, Wright J Fraser, Pierce Eric A, Testa Francesco, Mingozzi Federico, Bennicelli Jeannette L, Ying Gui-shuang, Rossi Settimio, Fulton Ann, Marshall Kathleen A, Banfi Sandro, Chung Daniel C, Morgan Jessica I W, Hauck Bernd, Zelenaia Olga, Zhu Xiaosong, Raffini Leslie, Coppieters Frauke, De Baere Elfride, Shindler Kenneth S, Volpe Nicholas J, Surace Enrico M, Acerra Carmela, Lyubarsky Arkady, Redmond T Michael, Stone Edwin, Sun Junwei, McDonnell Jennifer Wellman, Leroy Bart P, Simonelli Francesca, Bennett Jean

Abstract excerpt

BACKGROUND: Gene therapy has the potential to reverse disease or prevent further deterioration of vision in patients with incurable inherited retinal degeneration. We therefore did a phase 1 trial to assess the effect of gene therapy on retinal and visual function in children and adults with Leber's congenital amaurosis. METHODS: We assessed the retinal and visual function in 12 patients (aged 8-44 years) with...

Topics

Adolescent
Adult
Age Factors
Blindness
Carrier Proteins
Child
Dark Adaptation
Dependovirus

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Disease Progression

Dose-Response Relationship, Drug

Electroretinography

Eye Proteins

Female

Genetic Therapy

Genetic Vectors

Humans

Injections

Male

Source

Open in PubMed Open full text

Identifiers

PMID: 19854499
DOI: 10.1016/S0140-6736(09)61836-5
PMCID: PMC4492302

Genes

RPE65

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

Community interpretation

Discuss the evidence

Ask a question, add context or challenge an interpretation. Community activity is separate from scientific evidence strength.

discussions

replies

Citations encouraged

Claims remain attributable.

No discussion yet

Be the first to add a careful, cited interpretation.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.PMID 19854499Read ↗

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 ↗

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

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

Effect of gene therapy on visual function in Leber's congenital amaurosis.Semantic proximity Read ↗

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

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

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 ↗

Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65Semantic proximity Read ↗

Leber congenital amaurosis/early-onset severe retinal dystrophy: current management and clinical trials.Semantic proximity Read ↗

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

Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital AmaurosisSemantic proximity Read ↗

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