Article
Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice.
Nature biotechnology - 1 Oct 2018
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
Abstract excerpt
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 tyrosinemia type I and mucopolysaccharidosis type I. This approach recombines non-mutated genetic information present in...
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