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Exon-Skipping Oligonucleotides Restore Functional Collagen VI by Correcting a Common COL6A1 Mutation in Ullrich CMD
by
Aguti, Sara
,
Bolduc, Véronique
,
Ala, Pierpaolo
,
Turmaine, Mark
,
Bönnemann, Carsten G.
,
Muntoni, Francesco
,
Zhou, Haiyan
Published in
Molecular therapy. Nucleic acids
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Novel Biomarkers for Limb Girdle Muscular Dystrophy (LGMD)
by
Aguti, Sara
,
Gallus, Gian Nicola
,
Bianchi, Silvia
,
Salvatore, Simona
,
Rubegni, Anna
,
Berti, Gianna
,
Formichi, Patrizia
,
De Stefano, Nicola
,
Malandrini, Alessandro
,
Lopergolo, Diego
Published in
Cells (Basel, Switzerland)
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Strategies to improve the design of gapmer antisense oligonucleotide on allele-specific silencing
by
Aguti, Sara
,
Cheng, Shuzhi
,
Ala, Pierpaolo
,
Briggs, Sean
,
Muntoni, Francesco
,
Zhou, Haiyan
Published in
Molecular therapy. Nucleic acids
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A recurrent COL6A1 pseudoexon insertion causes muscular dystrophy and is effectively targeted by splice-correction therapies
by
Bolduc, Véronique
,
Foley, A Reghan
,
Solomon-Degefa, Herimela
,
Sarathy, Apurva
,
Donkervoort, Sandra
,
Hu, Ying
,
Chen, Grace S
,
Sizov, Katherine
,
Nalls, Matthew
,
Zhou, Haiyan
,
Aguti, Sara
,
Cummings, Beryl B
,
Lek, Monkol
,
Tukiainen, Taru
,
Marshall, Jamie L
,
Regev, Oded
,
Marek-Yagel, Dina
,
Sarkozy, Anna
,
Butterfield, Russell J
,
Jou, Cristina
,
Jimenez-Mallebrera, Cecilia
,
Li, Yan
,
Gartioux, Corine
,
Mamchaoui, Kamel
,
Allamand, Valérie
,
Gualandi, Francesca
,
Ferlini, Alessandra
,
Hanssen, Eric
,
Wilton, Steve D
,
Lamandé, Shireen R
,
MacArthur, Daniel G
,
Wagener, Raimund
,
Muntoni, Francesco
,
Bönnemann, Carsten G
Published in
JCI insight
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Molecular Therapy. Nucleic Acids
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Cells
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Life Sciences & Biomedicine
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Science & Technology
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Medicine, Research & Experimental
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Antisense Oligonucleotide
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Collagen Vi
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Allele-Specific Silencing
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Base Sequence
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Capn3
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Collagen Type Vi - Genetics
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