Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment-Reference-Cited by-同舟云学术

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Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment

Published:2017-06 Issue: Volume:7 Page:81-89
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Robin Valérie,Griffith Graziella,Carter John-Paul L.,Leumann Christian J.,Garcia Luis,Goyenvalle Aurélie

Funder

Agence Nationale de la Recherche

Chair of Excellence HandiMedEx

Association Monegasque contre les Myopathies

Duchenne Parent Project France

Institut National de la Santé et de la Recherche Médicale (INSERM)

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference48 articles.

1. Identification and characterization of a spinal muscular atrophy-determining gene;Lefebvre;Cell,1995

2. A novel nuclear structure containing the survival of motor neurons protein;Liu;EMBO J.,1996

3. Essential role for the SMN complex in the specificity of snRNP assembly;Pellizzoni;Science,2002

4. Spinal muscular atrophy: the role of SMN in axonal mRNA regulation;Fallini;Brain Res.,2012

5. Quantitative analyses of SMN1 and SMN2 based on real-time lightCycler PCR: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy;Feldkötter;Am. J. Hum. Genet.,2002

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