RPE65-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy

BACKGROUND: This research aimed to establish recommendations on the clinical and genetic characteristics necessary to confirm patient eligibility for gene supplementation with voretigene neparvovec. METHODS: An expert steering committee comprising an interdisciplinary panel of Italian experts in the...

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Detaylı Bibliyografya
Yayımlandı:Orphanet J Rare Dis
Asıl Yazarlar: Sodi, Andrea, Banfi, Sandro, Testa, Francesco, Della Corte, Michele, Passerini, Ilaria, Pelo, Elisabetta, Rossi, Settimio, Simonelli, Francesca
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: BioMed Central 2021
Konular:
Research
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC8176684/
https://ncbi.nlm.nih.gov/pubmed/34088339
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-021-01868-4
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