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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Главные авторы: 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
Формат: Artigo
Язык:Inglês
Опубликовано: Oxford University Press 2009
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Articles
Online-ссылка: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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