Publications


A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model.
Mol Ther 2017 Sep 26;25(9):2038-2052. Epub 2017 Jun 26.
Centre of Research in Myology (CRM), Institut de Myologie, Sorbonne Universités, UPMC Univ Paris 06, Inserm UMRS974, GH Pitié Salpêtrière, Paris 75013, France.


Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice.
Hum Mol Genet 2011 Feb 30;20(4):681-93. Epub 2010 Nov 30.
UPMC-AIM UMR S974, INSERM U 974, CNRS UMR 7215, Institut de Myologie, Université Pierre et Marie Curie Paris 6, Paris, France.

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