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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: | , , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
2009
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| Fag: | |
| Online adgang: | 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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