Journal
NATURE BIOTECHNOLOGY
Volume 36, Issue 9, Pages 839-+Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/nbt.4219
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Funding
- National Institutes of Health [1P01AI100263, 1R01NS076991, 5P01HD080642, R01AI12135, DP2HL137167, P01HL131471]
- National High Technology Research and Development Program (863 Program) of China [2012AA020810]
- Hyundai Hope on Wheels
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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 two heterozygous alleles into one functional allele without using donor DNA templates.
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