Nature News

An impartial strategy to mutation for muscular dystrophy by way of the upregulation of a modifier gene


Okay. Gawlik, Y. Miyagoe-Suzuki, P. Ekblom, S. S. & Durbeej, M. The laminin chain reduces muscular dystrophy in mice poor in laminin α2 chain. Hum. Mol. Broom. 13, 1775-1784 (2004).


Kemaladewi, D. U. et al. Correction of a splice defect in a mouse mannequin of congenital muscular dystrophy sort 1A with the help of an impartial mechanism of homology, directed on the restore. Nat. Med. 23, 984-989 (2017).


Y. Sunada, S. Bernier, A. Utani, Yamada Y. and Campbell Okay. P. Identification of a brand new mutant transcript of the laminin α2 chain gene liable for muscular dystrophy and dysmyelination in mice
Mouse 2J. Hum. Mol. Broom. four, 1055-1061 (1995).


Gawlik, Okay.I., Harandi, V.M., Cheong, R. Y., Petersen, Å. & Durbeej, M. Laminin α1 Reduces Muscular Dystrophy in Males
Mouse 2J. Matrix Biol. 70, 36-49 (2018).


Gawlik, Okay.I., Li, J., Petersen, A. and Durbeej, M. The α1 laminin chain enhances α2-laminin poor peripheral chain neuropathy. Hum. Mol. Broom. 15, 2690-2700 (2006).


Maeder, M.L. et al. CRISPR RNA guided activation of endogenous human genes. Nat. Strategies 10, 977-979 (2013).


Perez-Pinera, P. et al. RNA-guided gene activation by transcription components based mostly on CRISPR-Cas9. Nat. Strategies 10, 973-976 (2013).


Wojtal, D. et al. Nature of spell checker: CRISPR / Cas9 versatility for the event of therapies for hereditary issues. A m. J. Hum. Broom. 98, 90-101 (2016).


Ran, F.A. et al. In vivo genome enhancing with the assistance of Staphylococcus aureus Cas9. Nature 520, 186-191 (2015).