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Current Progress in Therapeutic Gene Editing for Monogenic Diseases

Programmable nucleases allow defined alterations in the genome with ease-of-use, efficiency, and specificity. Their availability has led to accurate and widespread genome engineering, with multiple applications in basic research, biotechnology, and therapy. With regard to human gene therapy, nucleas...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Prakash, Versha, Moore, Marc, Yáñez-Muñoz, Rafael J
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4786935/
https://ncbi.nlm.nih.gov/pubmed/26765770
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.5
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