ArticleNature biotechnology2018
Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice.
Article in Nature biotechnology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers.
What it found
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The trial behind it
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Who cites it
31 citing papers in PubMed.
- Donor-complementary prime editing enables precise kilobase and library-compatible DNA insertions.Nature biotechnology · 2026Article
- CRISPR/Cas9-mediated gene correction of Wilson disease H1069Q point mutation in patient-specific induced pluripotent stem cells.Gene therapy · 2026Article
- High-accuracy crRNA array assembly strategy for multiplex CRISPR.Molecular therapy. Nucleic acids · 2025Article
- AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys.Human gene therapy · 2024Article
- Viral and nonviral nanocarriers forNano research · 2024Article
- Targeted genome editing restores auditory function in adult mice with progressive hearing loss caused by a human microRNA mutation.Science translational medicine · 2024Article
- AAV-based gene editing of type 1 collagen mutation to treat osteogenesis imperfecta.Molecular therapy. Nucleic acids · 2024Article
- A Fluorescent Reporter Mouse forThe CRISPR journal · 2023Article
- CRISPR/Cas9 Genome Editing for Tissue-Specific In Vivo Targeting: Nanomaterials and Translational Perspective.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2023Review
- Genome Editing Tools for Lysosomal Storage Disorders.Advances in experimental medicine and biology · 2023Article
- Site-specific genome editing in treatment of inherited diseases: possibility, progress, and perspectives.Medical review (2021) · 2022Review
- Article
- Versatile and efficient in vivo genome editing with compact Streptococcus pasteurianus Cas9.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo.Nature communications · 2021Article
- Evolving AAV-delivered therapeutics towards ultimate cures.Journal of molecular medicine (Berlin, Germany) · 2021Review
- Approach for in vivo delivery of CRISPR/Cas system: a recent update and future prospect.Cellular and molecular life sciences : CMLS · 2021Review
- POLQ suppresses interhomolog recombination and loss of heterozygosity at targeted DNA breaks.Proceedings of the National Academy of Sciences of the United States of America · 2020Article
- Genome Editing for Rare Diseases.Current stem cell reports · 2020Article
- Translating CRISPR-Cas Therapeutics: Approaches and Challenges.The CRISPR journal · 2020Review
Corrections and comments
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Authors and funding
15 authors.
Funding
Abstract
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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Registered trials
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