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Gene therapy for metabolic diseases

Gene therapy has recently shown great promise as an effective treatment for a number of metabolic diseases caused by genetic defects in both animal models and human clinical trials. Most of the current success has been achieved using a viral mediated gene addition approach, but gene-editing technolo...

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Detalles Bibliográficos
Autores principales: Chandler, Randy J., Venditti, Charles P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5108627/
https://www.ncbi.nlm.nih.gov/pubmed/27853673
http://dx.doi.org/10.3233/TRD-160007