Correction of the Marfan Syndrome Pathogenic FBN1 Mutation by Base Editing in Human Cells and Heterozygous Embryos

There are urgent demands for efficient treatment of heritable genetic diseases. The base editing technology has displayed its efficiency and precision in base substitution in human embryos, providing a potential early-stage treatment for genetic diseases. Taking advantage of this technology, we corr...

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Detalles Bibliográficos
Autores principales: Zeng, Yanting, Li, Jianan, Li, Guanglei, Huang, Shisheng, Yu, Wenxia, Zhang, Yu, Chen, Dunjin, Chen, Jia, Liu, Jianqiao, Huang, Xingxu
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6224777/
https://www.ncbi.nlm.nih.gov/pubmed/30166242
http://dx.doi.org/10.1016/j.ymthe.2018.08.007

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6224777/
https://www.ncbi.nlm.nih.gov/pubmed/30166242
http://dx.doi.org/10.1016/j.ymthe.2018.08.007

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