Suppression and Replacement Gene Therapy for Autosomal Dominant Disease in a Murine Model of Dominant Retinitis Pigmentosa

For dominantly inherited disorders development of gene therapies, targeting the primary genetic lesion has been impeded by mutational heterogeneity. An example is rhodopsin-linked autosomal dominant retinitis pigmentosa with over 150 mutations in the rhodopsin gene. Validation of a mutation-independ...

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Autors principals: Millington-Ward, Sophia, Chadderton, Naomi, O'Reilly, Mary, Palfi, Arpad, Goldmann, Tobias, Kilty, Claire, Humphries, Marian, Wolfrum, Uwe, Bennett, Jean, Humphries, Peter, Kenna, Paul F, Farrar, G Jane
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2011
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3070095/
https://ncbi.nlm.nih.gov/pubmed/21224835
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.293
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