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In vivo adenine base editing corrects newborn murine model of Hurler syndrome
Mucopolysaccharidosis type I (MPS I) is a severe disease caused by loss-of-function mutation variants in the α-L-iduronidase (Idua) gene. In vivo genome editing represents a promising strategy to correct Idua mutations, and has the potential to permanently restore IDUA function over the lifespan of...
Autores principales: | Su, Jing, Jin, Xiu, She, Kaiqin, Liu, Yi, Song, Li, Zhao, Qinyu, Xiao, Jianlu, Li, Ruiting, Deng, Hongxin, Lu, Fang, Yang, Yang |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer Nature Singapore
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9947215/ https://www.ncbi.nlm.nih.gov/pubmed/36813914 http://dx.doi.org/10.1186/s43556-023-00120-8 |
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