A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene-Reference-Cited by-同舟云学术

A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene

Published:2019-07-24 Issue:7767 Volume:572 Page:125-130
ISSN:0028-0836
Container-title:Nature
language:en
Short-container-title:Nature

Author:

Kemaladewi Dwi U.,Bassi Prabhpreet S.,Erwood Steven,Al-Basha Dhekra,Gawlik Kinga I.,Lindsay Kyle,Hyatt Elzbieta,Kember Rebekah,Place Kara M.,Marks Ryan M.,Durbeej Madeleine,Prescott Steven A.,Ivakine Evgueni A.,Cohn Ronald D.

Publisher

Springer Science and Business Media LLC

Subject

Multidisciplinary

Link

http://www.nature.com/articles/s41586-019-1430-x.pdf

Reference27 articles.

1. Gawlik, K., Miyagoe-Suzuki, Y., Ekblom, P., Takeda, S. & Durbeej, M. Laminin α1 chain reduces muscular dystrophy in laminin α2 chain deficient mice. Hum. Mol. Genet. 13, 1775–1784 (2004).

2. Kemaladewi, D. U. et al. Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism. Nat. Med. 23, 984–989 (2017).

3. Sunada, Y., Bernier, S. M., Utani, A., Yamada, Y. & Campbell, K. P. Identification of a novel mutant transcript of laminin α2 chain gene responsible for muscular dystrophy and dysmyelination in dy 2J mice. Hum. Mol. Genet. 4, 1055–1061 (1995).

4. Gawlik, K. I., Harandi, V. M., Cheong, R. Y., Petersén, Å. & Durbeej, M. Laminin α1 reduces muscular dystrophy in dy 2J mice. Matrix Biol. 70, 36–49 (2018).

5. Gawlik, K. I., Li, J. Y., Petersén, A. & Durbeej, M. Laminin α1 chain improves laminin α2 chain deficient peripheral neuropathy. Hum. Mol. Genet. 15, 2690–2700 (2006).

Cited by 108 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Genetic therapies and potential therapeutic applications of CRISPR activators in the eye;Progress in Retinal and Eye Research;2024-09

2. An International Retrospective Early Natural History Study of LAMA2-Related Dystrophies;Journal of Neuromuscular Diseases;2024-08-22

3. Precision epigenetic editing: Technological advances, enduring challenges, and therapeutic applications;Cell Chemical Biology;2024-08

4. On RNA-programmable gene modulation as a versatile set of principles targeting muscular dystrophies;Molecular Therapy;2024-08

5. Long term rescue of Alzheimer’s deficitsin vivoby one-time gene-editing ofAppC-terminus;2024-06-09