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Spotlight on childhood blindness
Leber congenital amaurosis (LCA) is a rare disease that severely affects vision in early life. It is characterized by genetic and clinical heterogeneity due to complex and not fully understood pathogenetic mechanisms. It is also now widely known as a disease model for gene therapy. In this issue of...
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
American Society for Clinical Investigation
2011
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| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3104785/ https://ncbi.nlm.nih.gov/pubmed/21606601 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI58300 |
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