DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice

Mutations in the photoreceptor-specific flippase ABCA4 are associated with Stargardt disease and many other forms of retinal degeneration that currently lack curative therapies. Gene replacement is a logical strategy for ABCA4-associated disease, particularly given the current success of traditional...

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Detaylı Bibliyografya
Asıl Yazarlar: Han, Zongchao, Conley, Shannon M., Makkia, Rasha S., Cooper, Mark J., Naash, Muna I.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: American Society for Clinical Investigation 2012
Konular:
Brief Report
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC3428101/
https://ncbi.nlm.nih.gov/pubmed/22886305
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI64833
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