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...

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Detalles Bibliográficos
Autores principales: Su, Jing, Jin, Xiu, She, Kaiqin, Liu, Yi, Song, Li, Zhao, Qinyu, Xiao, Jianlu, Li, Ruiting, Deng, Hongxin, Lu, Fang, Yang, Yang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Nature Singapore 2023
Materias:
Research
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

Internet

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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