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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...
Autores principales: | , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
2018
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Materias: | |
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 |