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In vivo gene editing in dystrophic mouse muscle and muscle stem cells.

Tabebordbar M, Zhu K, Cheng JK, Chew WL, Widrick JJ, Yan WX, Maesner C, Wu EY, Xiao R, Ran FA, Cong L, Zhang F, Vandenberghe LH, Church GM, Wagers AJ.

Science. 2016 Jan 22;351(6271):407-11. doi: 10.1126/science.aad5177. Epub 2015 Dec 31.


Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice.

Gao X, Shen X, Dong X, Ran N, Han G, Cao L, Gu B, Yin H.

Mol Ther Nucleic Acids. 2015 Oct 6;4:e255. doi: 10.1038/mtna.2015.27.


Aquapuncture Using Stem Cell Therapy to Treat Mdx Mice.

Esper GV, Pignatari GC, Rodrigues MN, Bertagnon HG, Fernandes IR, Nascimento N, Tabosa AM, Beltrão-Braga PC, Miglino MA.

Evid Based Complement Alternat Med. 2015;2015:132706. doi: 10.1155/2015/132706. Epub 2015 May 13.


X-Linked Dilated Cardiomyopathy: A Cardiospecific Phenotype of Dystrophinopathy.

Nakamura A.

Pharmaceuticals (Basel). 2015 Jun 9;8(2):303-20. doi: 10.3390/ph8020303. Review.


Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy.

McGreevy JW, Hakim CH, McIntosh MA, Duan D.

Dis Model Mech. 2015 Mar;8(3):195-213. doi: 10.1242/dmm.018424. Review.


High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomics.

Gintjee TJ, Magh AS, Bertoni C.

Biology (Basel). 2014 Nov 14;3(4):752-80. doi: 10.3390/biology3040752. Review.


Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in Duchenne muscular dystrophy.

Cao L, Han G, Gu B, Yin H.

PLoS One. 2014 Nov 3;9(11):e111079. doi: 10.1371/journal.pone.0111079. eCollection 2014.


Antisense-mediated Exon Skipping Decreases Tau Protein Expression: A Potential Therapy For Tauopathies.

Sud R, Geller ET, Schellenberg GD.

Mol Ther Nucleic Acids. 2014 Jul 29;3:e180. doi: 10.1038/mtna.2014.30.


Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice.

Wu B, Cloer C, Lu P, Milazi S, Shaban M, Shah SN, Marston-Poe L, Moulton HM, Lu QL.

Gene Ther. 2014 Sep;21(9):785-93. doi: 10.1038/gt.2014.53. Epub 2014 Jun 19.


Nanoparticle delivery of antisense oligonucleotides and their application in the exon skipping strategy for Duchenne muscular dystrophy.

Falzarano MS, Passarelli C, Ferlini A.

Nucleic Acid Ther. 2014 Feb;24(1):87-100. doi: 10.1089/nat.2013.0450. Review.


Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases.

Siva K, Covello G, Denti MA.

Nucleic Acid Ther. 2014 Feb;24(1):69-86. doi: 10.1089/nat.2013.0461. Review.


Antisense mediated splicing modulation for inherited metabolic diseases: challenges for delivery.

Pérez B, Vilageliu L, Grinberg D, Desviat LR.

Nucleic Acid Ther. 2014 Feb;24(1):48-56. doi: 10.1089/nat.2013.0453. Review.


Cellular trafficking determines the exon skipping activity of Pip6a-PMO in mdx skeletal and cardiac muscle cells.

Lehto T, Castillo Alvarez A, Gauck S, Gait MJ, Coursindel T, Wood MJ, Lebleu B, Boisguerin P.

Nucleic Acids Res. 2014 Mar;42(5):3207-17. doi: 10.1093/nar/gkt1220. Epub 2013 Dec 23.


Morpholino treatment improves muscle function and pathology of Pitx1 transgenic mice.

Pandey SN, Lee YC, Yokota T, Chen YW.

Mol Ther. 2014 Feb;22(2):390-6. doi: 10.1038/mt.2013.263. Epub 2013 Nov 14.


Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches.

Lorain S, Peccate C, Le Hir M, Griffith G, Philippi S, Précigout G, Mamchaoui K, Jollet A, Voit T, Garcia L.

Nucleic Acids Res. 2013 Sep;41(17):8391-402. doi: 10.1093/nar/gkt621. Epub 2013 Jul 16.


Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agents.

Wilton SD, Fletcher S.

Appl Clin Genet. 2011 Mar 10;4:29-44. doi: 10.2147/TACG.S8762. Print 2011.


Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice.

Gedicke-Hornung C, Behrens-Gawlik V, Reischmann S, Geertz B, Stimpel D, Weinberger F, Schlossarek S, Précigout G, Braren I, Eschenhagen T, Mearini G, Lorain S, Voit T, Dreyfus PA, Garcia L, Carrier L.

EMBO Mol Med. 2013 Jul;5(7):1060-77. doi: 10.1002/emmm.201202168. Epub 2013 May 29.


Effective exon skipping and dystrophin restoration by 2'-o-methoxyethyl antisense oligonucleotide in dystrophin-deficient mice.

Yang L, Niu H, Gao X, Wang Q, Han G, Cao L, Cai C, Weiler J, Yin H.

PLoS One. 2013 Apr 26;8(4):e61584. doi: 10.1371/journal.pone.0061584. Print 2013.


Zebrafish based small molecule screens for novel DMD drugs.

Kawahara G, Kunkel LM.

Drug Discov Today Technol. 2013 Spring;10(1):e91-e96.


Splicing therapy for neuromuscular disease.

Douglas AG, Wood MJ.

Mol Cell Neurosci. 2013 Sep;56:169-85. doi: 10.1016/j.mcn.2013.04.005. Epub 2013 Apr 28. Review.

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