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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Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Oxford University Press 2009
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On-line přístup: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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