Evaluating the Impact of Variable Phosphorothioate Content in Tricyclo-DNA Antisense Oligonucleotides in a Duchenne Muscular Dystrophy Mouse Model

Lucía Echevarría,Philippine Aupy,Karima Relizani,Thomas Bestetti,Graziella Griffith,Florence Blandel,Marek Komisarski,Adrian Haeberli,Fedor Svinartchouk,Luis Garcia,Aurélie Goyenvalle

Evaluating the Impact of Variable Phosphorothioate Content in Tricyclo-DNA Antisense Oligonucleotides in a Duchenne Muscular Dystrophy Mouse Model

2019

Lucía Echevarría
Philippine Aupy
Karima Relizani
Thomas Bestetti
Graziella Griffith
Florence Blandel
Marek Komisarski
Adrian Haeberli
Fedor Svinartchouk
Luis Garcia
Aurélie Goyenvalle

Antisense oligonucleotides (ASOs) hold promise for therapeutic splice switching correction for genetic diseases, in particular for Duchenne muscular dystrophy (DMD), for which ASO-exon skipping rep...

Keywords:

Duchenne muscular dystrophy
Oligonucleotide
DNA
Sense (molecular biology)
Biochemistry
Biology
Molecular biology
antisense oligonucleotides
splice switching
RNA splicing

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