What are the latest recommended treatment options for Leber congenital amaurosis?

Gene therapies offer targeted treatment options for Leber congenital amaurosis, with Luxturna being the approved treatment for RPE65 mutations and various emerging therapies under investigation for CEP290 and other subtypes. Each therapy's application depends on specific genetic mutations, emphasizing the importance of genetic testing in management decisions.

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Publication Details

Journal
Zenodo (CERN European Organization for Nuclear Research)
Published
2026-11-04
DOI
https://doi.org/10.5281/zenodo.23956354
Primary Topic
Retinal Development and Disorders
Type
article
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article

What are the latest recommended treatment options for Leber congenital amaurosis?

Tripdatabase
Zenodo (CERN European Organization for Nuclear Research)
Retinal Development and Disorders
article

What are the latest recommended treatment options for Leber congenital amaurosis?

Tripdatabase
article en

Abstract

Gene therapies offer targeted treatment options for Leber congenital amaurosis, with Luxturna being the approved treatment for RPE65 mutations and various emerging therapies under investigation for CEP290 and other subtypes. Each therapy's application depends on specific genetic mutations, emphasizing the importance of genetic testing in management decisions.

Zenodo (CERN European Organization for Nuclear Research)
Openalex Percentile: Top 25%
Retinal Development and Disorders
DOIOpen Access PDFOpenAlex
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What are the latest recommended treatment options for Leber congenital amaurosis? — Tripdatabase · Zenodo (CERN European Organization for Nuclear Research) (2026) | TGRS Research Map | TGRS