Efficient Downregulation of <i>Alk4</i> in Skeletal Muscle After Systemic Treatment with Conjugated siRNAs in a Mouse Model for Duchenne Muscular Dystrophy-Reference-Cited by-同舟云学术

Efficient Downregulation of Alk4 in Skeletal Muscle After Systemic Treatment with Conjugated siRNAs in a Mouse Model for Duchenne Muscular Dystrophy

Published:2023-02-01 Issue:1 Volume:33 Page:26-34
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Engelbeen Sarah¹^ORCID,Pasteuning-Vuhman Svetlana¹,Boertje-van der Meulen Joke¹,Parmar Rubina²,Charisse Klaus²,Sepp-Lorenzino Laura²,Manoharan Muthiah²,Aartsma-Rus Annemieke¹^ORCID,van Putten Maaike¹

Affiliation:

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

2. Alnalym Pharmaceuticals, Cambridge, Massachusetts, USA.

Publisher

Mary Ann Liebert Inc

Subject

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

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2022.0021

Reference30 articles.

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

2. Innovative Therapeutic Approaches for Duchenne Muscular Dystrophy