Gene therapy for liver diseases — progress and challenges
Author:
Publisher
Springer Science and Business Media LLC
Subject
Gastroenterology,Hepatology
Link
https://www.nature.com/articles/s41575-022-00729-0.pdf
Reference253 articles.
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2. Wang, L. et al. AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice. Mol. Genet. Metab. 120, 299–305 (2017).
3. Unzu, C. et al. Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice. Mol. Ther. 19, 243–250 (2011).
4. Kaiser, R. A. et al. Use of an adeno-associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model. J. Inherit. Metab. Dis. 44, 1369–1381 (2021).
5. Weber, N. D. et al. Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model. Nat. Commun. 10, 5694 (2019).
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