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A single neonatal injection of an AAV9.U7snRNA virus mediating skipping of dmd exon 2 allows dystrophin expression preventing apparition of pathologic features in the Dup2 mouse one year post injection

Wein, N; Simmons, T; Gumienny, F; Huang, N; Heller, K; Yurkoski, J; Rodino-Klapac, L; ... Flanigan, K; + view all (2017) A single neonatal injection of an AAV9.U7snRNA virus mediating skipping of dmd exon 2 allows dystrophin expression preventing apparition of pathologic features in the Dup2 mouse one year post injection. Presented at: 22nd International Annual Congress of the World-Muscle-Society (WMS), Saint Malo, FRANCE.

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Type: Conference item (UNSPECIFIED)
Title: A single neonatal injection of an AAV9.U7snRNA virus mediating skipping of dmd exon 2 allows dystrophin expression preventing apparition of pathologic features in the Dup2 mouse one year post injection
Event: 22nd International Annual Congress of the World-Muscle-Society (WMS)
Location: Saint Malo, FRANCE
Dates: 03 October 2017 - 07 October 2017
DOI: 10.1016/j.nmd.2017.06.339
Keywords: Science & Technology, Life Sciences & Biomedicine, Clinical Neurology, Neurosciences, Neurosciences & Neurology
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Pop Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Pop Health Sciences > UCL GOS Institute of Child Health > ICH Developmental Neurosciences Prog
URI: http://discovery.ucl.ac.uk/id/eprint/10035090
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