Genome editing for inborn errors of metabolism: advancing towards the clinic

Inborn errors of metabolism (IEM) include many disorders for which current treatments aim to ameliorate disease manifestations, but are not curative. Advances in the field of genome editing have recently resulted in the in vivo correction of murine models of IEM. Site-specific endonucleases, such as...

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Detalles Bibliográficos
Autores principales: Schneller, Jessica L., Lee, Ciaran M., Bao, Gang, Venditti, Charles P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2017
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5327528/
https://www.ncbi.nlm.nih.gov/pubmed/28238287
http://dx.doi.org/10.1186/s12916-017-0798-4

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5327528/
https://www.ncbi.nlm.nih.gov/pubmed/28238287
http://dx.doi.org/10.1186/s12916-017-0798-4

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