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CRISPR-Cas9 correction in the DMD mouse model is accompanied by upregulation of Dp71f protein
Duchenne muscular dystrophy (DMD) is a severe hereditary disease caused by a deficiency in the dystrophin protein. The most frequent types of disease-causing mutations in the DMD gene are frameshift deletions of one or more exons. Precision genome editing systems such as CRISPR-Cas9 have shown poten...
Autores principales: | , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10339130/ https://www.ncbi.nlm.nih.gov/pubmed/37457303 http://dx.doi.org/10.1016/j.omtm.2023.06.006 |