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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...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Editorial Department of Journal of Biomedical Research
2021
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8038532/ https://www.ncbi.nlm.nih.gov/pubmed/33402545 http://dx.doi.org/10.7555/JBR.34.20200105 |