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CRISPR for Neuromuscular Disorders: Gene Editing and Beyond
This is a review describing advances in CRISPR/Cas-mediated therapies for neuromuscular disorders (NMDs). We explore both CRISPR-mediated editing and dead Cas approaches as potential therapeutic strategies for multiple NMDs. Last, therapeutic considerations, including delivery and off-target effects...
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| Publicado no: | Physiology (Bethesda) |
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| Main Authors: | , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Physiological Society
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6863376/ https://ncbi.nlm.nih.gov/pubmed/31389773 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1152/physiol.00012.2019 |
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