Diseases caused by different mutations in the two alleles of a gene are treated in mice by Cas-9-induced allelic exchange.: Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice

We report a genome-editing strategy to correct compound heterozygous mutations, a common genotype in patients with recessive genetic disorders. Adeno-associated viral vector delivery of Cas9 and guide RNA induces allelic exchange and rescues the disease phenotype in mouse models of hereditary tyrosi...

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Detalles Bibliográficos
Autores principales: Wang, Dan, Li, Jia, Song, Chun-Qing, Tran, Karen, Mou, Haiwei, Wu, Pei-Hsuan, Tai, Phillip W.L., Mendonca, Craig A., Ren, Lingzhi, Wang, Blake Y., Su, Qin, Gessler, Dominic J., Zamore, Phillip D., Xue, Wen, Gao, Guangping
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6126964/
https://www.ncbi.nlm.nih.gov/pubmed/30102296
http://dx.doi.org/10.1038/nbt.4219

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