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Which Leber congenital amaurosis patients are eligible for gene therapy trials?

BACKGROUND: In 2007, clinical trials began for gene-replacement therapy for RPE65-associated Leber congenital amaurosis (LCA). To enroll, subjects must have both disease-causing RPE65 alleles identified. Determining which patients have true disease-causing mutations requires a multistep approach. ME...

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Main Authors: Drack, Arlene V., Johnston, Rebecca, Stone, Edwin M.
格式: Artigo
語言:Inglês
出版: 2009
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC2860791/
https://ncbi.nlm.nih.gov/pubmed/19840725
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jaapos.2009.08.006
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