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Exon-skipping advances for Duchenne muscular dystrophy.
Echevarría L, Aupy P, Goyenvalle A. Echevarría L, et al. Among authors: aupy p. Hum Mol Genet. 2018 Aug 1;27(R2):R163-R172. doi: 10.1093/hmg/ddy171. Hum Mol Genet. 2018. PMID: 29771317 Review.
Palmitic acid conjugation enhances potency of tricyclo-DNA splice switching oligonucleotides.
Relizani K, Echevarría L, Zarrouki F, Gastaldi C, Dambrune C, Aupy P, Haeberli A, Komisarski M, Tensorer T, Larcher T, Svinartchouk F, Vaillend C, Garcia L, Goyenvalle A. Relizani K, et al. Among authors: aupy p. Nucleic Acids Res. 2022 Jan 11;50(1):17-34. doi: 10.1093/nar/gkab1199. Nucleic Acids Res. 2022. PMID: 34893881 Free PMC article.
Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice.
Aupy P, Zarrouki F, Sandro Q, Gastaldi C, Buclez PO, Mamchaoui K, Garcia L, Vaillend C, Goyenvalle A. Aupy P, et al. Mol Ther Methods Clin Dev. 2020 May 4;17:1037-1047. doi: 10.1016/j.omtm.2020.04.025. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32462052 Free PMC article.
Evaluating the Impact of Variable Phosphorothioate Content in Tricyclo-DNA Antisense Oligonucleotides in a Duchenne Muscular Dystrophy Mouse Model.
Echevarría L, Aupy P, Relizani K, Bestetti T, Griffith G, Blandel F, Komisarski M, Haeberli A, Svinartchouk F, Garcia L, Goyenvalle A. Echevarría L, et al. Among authors: aupy p. Nucleic Acid Ther. 2019 Jun;29(3):148-160. doi: 10.1089/nat.2018.0773. Epub 2019 Apr 22. Nucleic Acid Ther. 2019. PMID: 31009315