Enhanced Delivery of Ligand-Conjugated Antisense Oligonucleotides (C16-HA-ASO) Targeting Dystrophia Myotonica Protein Kinase Transcripts for the Treatment of Myotonic Dystrophy Type 1-Reference-Cited by-同舟云学术

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

Published:2022-08-01 Issue:15-16 Volume:33 Page:810-820
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Ait Benichou Siham¹,Jauvin Dominic¹²,De Serres-Bérard Thiéry¹²,Bennett Frank³,Rigo Frank³,Gourdon Genevieve⁴^ORCID,Boutjdir Mohamed⁵⁶⁷,Chahine Mohamed²⁸^ORCID,Puymirat Jack¹⁸

Affiliation:

1. LOEX, CHU de Québec-Université Laval Research Center, Quebec City, Canada.

2. CERVO Brain Research Center, Institut Universitaire en Santé Mentale de Québec, Quebec City, Canada.

3. Ionis Pharmaceuticals, Inc., Carlsbad, California, USA.

4. Centre de Recherche en Myologie, Association Institut de Myologie, Sorbonne Université, Inserm, Paris, France.

5. Cardiovascular Research Program, VA New York Harbor Healthcare System, Brooklyn, New York, USA.

6. Department of Medicine, Cell Biology, and Pharmacology, State University of New York Downstate Health Sciences University, Brooklyn, New York, USA.

7. Department of Medicine, NYU Grossman School of Medicine, New York, New York, USA.

8. Department of Medicine, Faculté de Médecine, Université Laval, Quebec City, Canada.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2022.069

Reference47 articles.

1. Molecular basis of myotonic dystrophy: Expansion of a trinucleotide (CTG) repeat at the 3′ end of a transcript encoding a protein kinase family member

2. An Unstable Triplet Repeat in a Gene Related to Myotonic Muscular Dystrophy

3. Myotonic Dystrophy Mutation: an Unstable CTG Repeat in the 3′ Untranslated region of the Gene

4. 4. Harper PS. Myotonic Dystrophy. W.B. Saunders: London, UK; 2001.

5. Severe cardiac arrhythmias in young patients with myotonic dystrophy type 1

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