Improved Retinal Function in a Mouse Model of Dominant Retinitis Pigmentosa Following AAV-delivered Gene Therapy

Mutational heterogeneity represents one of the greatest barriers impeding the progress toward the clinic of gene therapies for many dominantly inherited disorders. A general strategy of gene suppression in conjunction with replacement has been proposed to overcome this mutational heterogeneity. In t...

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Autori principali: Chadderton, Naomi, Millington-Ward, Sophia, Palfi, Arpad, O'Reilly, Mary, Tuohy, Gearóid, Humphries, Marian M, Li, Tiansen, Humphries, Peter, Kenna, Paul F, Farrar, G Jane
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2009
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Original Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835099/
https://ncbi.nlm.nih.gov/pubmed/19174761
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2008.301
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