Article
A mutation-independent CRISPR-Cas9-mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency.
Science advances - 1 Feb 2020
Wang Lili, Yang Yang, Breton Camilo, Bell Peter, Li Mingyao, Zhang Jia, Che Yan, Saveliev Alexei, He Zhenning, White John, Latshaw Caitlin, Xu Chenyu, McMenamin Deirdre, Yu Hongwei, Morizono Hiroki, Batshaw Mark L, Wilson James M
Abstract excerpt
Ornithine transcarbamylase (OTC) deficiency is an X-linked urea cycle disorder associated with high mortality. Although a promising treatment for late-onset OTC deficiency, adeno-associated virus (AAV) neonatal gene therapy would only provide short-term therapeutic effects as the non-integrated genome gets lost during hepatocyte proliferation. CRISPR-Cas9-mediated homology-directed repair can correct a G-to-A...
Topics
- Animals
- CRISPR-Cas Systems
- DNA Repair
- Dependovirus
- Dietary Proteins
- Disease Models, Animal
- Gene Targeting
- Genetic Loci
- Genetic Therapy
- Genetic Vectors