Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping
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Published in Molecular therapy. Methods & clinical development (11.06.2021)
Published in Molecular therapy. Methods & clinical development (11.06.2021)
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Current Status of Antisense Oligonucleotide-Based Therapy in Neuromuscular Disorders
Bizot, Flavien, Vulin, Adeline, Goyenvalle, Aurélie
Published in Drugs (New York, N.Y.) (01.09.2020)
Published in Drugs (New York, N.Y.) (01.09.2020)
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505. Treatment of DMD 5’ Mutations Through Two Different exon2 Skipping Strategies: Intramuscular Delivery of rAAV9.snRNA Mediated Skipping and Antisense Morpholino Oligomers
Simmons, Tabatha R., Wein, Nicolas, Vulin-Chaffiol, Adeline, Heller, Kristin, Rutherford, Andrea, Rodino-Klapac, Louise, Flanigan, Kevin M.
Published in Molecular therapy (01.05.2015)
Published in Molecular therapy (01.05.2015)
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