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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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書誌詳細
主要な著者: Cideciyan, Artur V., Swider, Malgorzata, Aleman, Tomas S., Tsybovsky, Yaroslav, Schwartz, Sharon B., Windsor, Elizabeth A.M., Roman, Alejandro J., Sumaroka, Alexander, Steinberg, Janet D., Jacobson, Samuel G., Stone, Edwin M., Palczewski, Krzysztof
フォーマット: Artigo
言語:Inglês
出版事項: Oxford University Press 2009
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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