Enhanced Delivery of Ligand-Conjugated Antisense Oligonucleotides (C16-HA-ASO) Targeting Dystrophia Myotonica Protein Kinase Transcripts for the Treatment of Myotonic Dystrophy Type 1 - Sorbonne Université Access content directly
Journal Articles Human Gene Therapy Year : 2022

Enhanced Delivery of Ligand-Conjugated Antisense Oligonucleotides (C16-HA-ASO) Targeting Dystrophia Myotonica Protein Kinase Transcripts for the Treatment of Myotonic Dystrophy Type 1

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https://hal.sorbonne-universite.fr/hal-03753318

Submitted on : Thursday, August 18, 2022-10:33:25 AM

Last modification on : Saturday, October 7, 2023-9:36:25 PM

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hal-03753318 , version 1 (18-08-2022)

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Siham Ait Benichou, Dominic Jauvin, Thiéry de Serres-Bérard, Frank Bennett, Frank Rigo, et al.. Enhanced Delivery of Ligand-Conjugated Antisense Oligonucleotides (C16-HA-ASO) Targeting Dystrophia Myotonica Protein Kinase Transcripts for the Treatment of Myotonic Dystrophy Type 1. Human Gene Therapy, 2022, 33 (15-16), pp.810-820. ⟨10.1089/hum.2022.069⟩. ⟨hal-03753318⟩

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