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).
Kemaladewi, D. U., Benjamin, J.S., Hyatt E., Ivakine E., A.A. and Cohn, R.D. Elevated ranges of polyamines as protecting modifiers of the illness in congenital muscular dystrophy. Hum. Mol. Broom. 27, 1905-1912 (2018).
Bönnemann, C.G. et al. Diagnostic strategy of congenital muscular dystrophies. Neuromuscul. Dysfunction. 24, 289-311 (2014).
Homma, S., Beermann, M.L. and Miller, J. B. Peripheral nerve pathology, together with differentiation of aberrant Schwann cells, is enhanced by doxycycline in a murine mannequin of congenital muscular dystrophy poor in laminin-α2. Hum. Mol. Broom. 20, 2662-2672 (2011).
Qiao, C. et al. Enchancment of muscular and nervous pathology in LAMA2 muscular dystrophy by AAV9-mini-agrin. Mol. Ther. Strategies Clin. Dev. 9, 47-56 (2018).
Patton, B.L., Wang, B., Y.S., Seburn, Okay.L. & Burgess, R.W. Some extent mutation within the LN area of LAMA2 causes muscular dystrophy and peripheral amyelinization. J. Cell Sci. 121, 1593-1604 (2008).
Previtali, S.C. et al. Expression of Laminin Receptors in Schwann Cell Differentiation: Proof of Distinct Roles. J. Neurosci. 23, 5520-5530 (2003).
Bentzinger, C.F., Barzaghi, P., Lin, S. & Ruegg, A. Overexpression of mini-agrin in skeletal muscle enhances muscle integrity and regenerative capability in α-laminin poor mice. FASEB J. 19, 934-942 (2005).
Reinhard, J.R. et al. Binding proteins restore the basement membrane and proper LAMA2-related muscular dystrophy in mice. Sci. Trad. Med. 9, eaal4649 (2017).
McKee, Okay. Okay. et al. The restore of chimeric proteins in laminin polymerization improves the phenotype of muscular dystrophy. J. Clin. Make investments. 127, 1075-1089 (2017).
Rooney, J.E., Gurpur, P.B. & Burkin, D.J.Laminin-111 protein remedy prevents muscle illnesses in mdx mice of Duchenne muscular dystrophy. Proc. Natl Acad. Sci. USA 106, 7991-7996 (2009).
Perrin, A., Rousseau, J. and Tremblay, J. P. Elevated expression of the alpha 1 chain of laminin by dCas9 – VP160. Mol. Ther. Nucleic Acids 6, 68-79 (2017).
Yuan, J. et al. Genetic modulation of RNA splicing with a CRISPR guided cytidine deaminase. Mol Cell 72, 380-394 (2018).
Villiger, L. et al. Remedy of metabolic liver illness by in vivo enhancing of the genome base in grownup mice. Nat. Med. 24, 1519-1525 (2018).
Bengtsson, N.E. et al. Modification of the muscle-specific dystrophin CRISPR / Cas9 gene enhances pathophysiology in a mouse mannequin of Duchenne muscular dystrophy. Nat. Widespread. eight, 14454 (2017).
Liao, H. Okay. et al. In vivo activation of the goal gene by trans-epigenetic modulation mediated by CRISPR / Cas9. Cell 171, 1495-1507 (2017).
Thakore, P.I. et al. Extremely particular epigenome edited by CRISPR – Cas9 repressors to silence distal regulatory parts. Nat. Strategies 12, 1143-1149 (2015).
Zhang, Y. et al. Notch1 signaling performs a task within the regulation of precursor differentiation throughout remyelination of the CNS. Proc. Natl Acad. Sci. USA 106, 19162-19167 (2009).
Hakim, C.H. et al. An improved dystrophic phenotype of the five-repeat micro-dystrophin gene within the extreme DBA / 2J-mdx mannequin of Duchenne muscular dystrophy. Mol. Ther. Strategies Clin. Dev. 6, 216-230 (2017).