Successful Gene Therapy in the RPGRIP1-deficient Dog: a Large Model of Cone–Rod Dystrophy

For the development of new therapies, proof-of-concept studies in large animal models that share clinical features with their human counterparts represent a pivotal step. For inherited retinal dystrophies primarily involving photoreceptor cells, the efficacy of gene therapy has been demonstrated in...

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Autori principali: Lhériteau, Elsa, Petit, Lolita, Weber, Michel, Le Meur, Guylène, Deschamps, Jack-Yves, Libeau, Lyse, Mendes-Madeira, Alexandra, Guihal, Caroline, François, Achille, Guyon, Richard, Provost, Nathalie, Lemoine, Françoise, Papal, Samantha, El-Amraoui, Aziz, Colle, Marie-Anne, Moullier, Philippe, Rolling, Fabienne
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2014
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3918913/
https://ncbi.nlm.nih.gov/pubmed/24091916
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.232
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