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Progress and challenges in CRISPR-mediated therapeutic genome editing for monogenic diseases

There are an estimated 10 000 monogenic diseases affecting tens of millions of individuals worldwide. The application of CRISPR/Cas genome editing tools to treat monogenic diseases is an emerging strategy with the potential to generate personalized treatment approaches for these patients. CRISPR/Cas...

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Detalles Bibliográficos
Publicado en:J Biomed Res
Autores principales: Konishi, Colin T., Long, Chengzu
Formato: Artigo
Lenguaje:Inglês
Publicado: Editorial Department of Journal of Biomedical Research 2021
Materias:
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC8038532/
https://ncbi.nlm.nih.gov/pubmed/33402545
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.7555/JBR.34.20200105
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