Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism-Reference-Cited by-同舟云学术

Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism

Published:2017-07-17 Issue:8 Volume:23 Page:984-989
ISSN:1078-8956
Container-title:Nature Medicine
language:en
Short-container-title:Nat Med

Author:

Kemaladewi Dwi U,Maino Eleonora,Hyatt Elzbieta,Hou Huayun,Ding Maylynn,Place Kara M,Zhu Xinyi,Bassi Prabhpreet,Baghestani Zahra,Deshwar Amit G,Merico Daniele,Xiong Hui Y,Frey Brendan J,Wilson Michael D^ORCID,Ivakine Evgueni A,Cohn Ronald D

Publisher

Springer Science and Business Media LLC

Subject

General Biochemistry, Genetics and Molecular Biology,General Medicine

Link

http://www.nature.com/articles/nm.4367.pdf

Reference40 articles.

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2. Bladen, C.L. et al. The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations. Hum. Mutat. 36, 395–402 (2015).

3. Yang, Y. et al. A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice. Nat. Biotechnol. 34, 334–338 (2016).

4. Yin, H. et al. Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nat. Biotechnol. 32, 551–553 (2014).

5. Yin, H. et al. Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo. Nat. Biotechnol. 34, 328–333 (2016).

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