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Page 1
Exon-skipping advances for Duchenne muscular dystrophy.
Hum Mol Genet. 2018 Aug 1;27(R2):R163-R172. doi: 10.1093/hmg/ddy171.
Hum Mol Genet. 2018.
PMID: 29771317
Review.
The Use of Tricyclo-DNA Oligomers for the Treatment of Genetic Disorders.
Aupy P, Echevarría L, Relizani K, Goyenvalle A.
Aupy P, et al.
Biomedicines. 2017 Dec 22;6(1):2. doi: 10.3390/biomedicines6010002.
Biomedicines. 2017.
PMID: 29271929
Free PMC article.
Review.
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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.
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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.
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Identifying and Avoiding tcDNA-ASO Sequence-Specific Toxicity for the Development of DMD Exon 51 Skipping Therapy.
Aupy P, Echevarría L, Relizani K, Zarrouki F, Haeberli A, Komisarski M, Tensorer T, Jouvion G, Svinartchouk F, Garcia L, Goyenvalle A.
Aupy P, et al.
Mol Ther Nucleic Acids. 2020 Mar 6;19:371-383. doi: 10.1016/j.omtn.2019.11.020. Epub 2019 Nov 27.
Mol Ther Nucleic Acids. 2020.
PMID: 31881528
Free PMC article.
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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
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