Challenges of Assessing Exon 53 Skipping of the Human <i>DMD</i> Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy-Reference-Cited by-同舟云学术

Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy

Published:2023-12-01 Issue:6 Volume:33 Page:348-360
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Engelbeen Sarah¹^ORCID,O'Reilly Daniel²³,Van De Vijver Davy¹,Verhaart Ingrid¹^ORCID,van Putten Maaike¹,Hariharan Vignesh²,Hassler Matthew²,Khvorova Anastasia²,Damha Masad J.³,Aartsma-Rus Annemieke¹^ORCID

Affiliation:

1. Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.

2. University of Massachusetts Chan Medical School, RNA Therapeutics Institute, Worcester, Massachusetts, USA.

3. Department of Chemistry, McGill University, Montreal, Canada.

Publisher

Mary Ann Liebert Inc

Subject

Drug Discovery,Genetics,Molecular Biology,Molecular Medicine,Biochemistry

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2023.0038

Reference32 articles.

1. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

2. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

3. Dystrophin: The protein product of the duchenne muscular dystrophy locus

4. Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update

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