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ABCA4 disease progression and a proposed strategy for gene therapy
Autosomal recessive retinal diseases caused by mutations in the ABCA4 gene are being considered for gene replacement therapy. All individuals with ABCA4-disease show macular degeneration, but only some are thought to progress to retina-wide blindness. It is currently not predictable if or when speci...
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主要な著者: | , , , , , , , , , , , |
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フォーマット: | Artigo |
言語: | Inglês |
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Oxford University Press
2009
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主題: | |
オンライン・アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2640207/ https://ncbi.nlm.nih.gov/pubmed/19074458 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddn421 |
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