Allele-Selective LNA Gapmers for the Treatment of Fibrodysplasia Ossificans Progressiva Knock Down the Pathogenic ACVR1<sup>R206H</sup> Transcript and Inhibit Osteogenic Differentiation-Reference-Cited by-同舟云学术

Allele-Selective LNA Gapmers for the Treatment of Fibrodysplasia Ossificans Progressiva Knock Down the Pathogenic ACVR1^R206H Transcript and Inhibit Osteogenic Differentiation

Published:2022-06-01 Issue:3 Volume:32 Page:185-193
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Maruyama Rika¹^ORCID,Nguyen Quynh¹,Roshmi Rohini Roy¹,Touznik Aleksander¹,Yokota Toshifumi¹²

Affiliation:

1. Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, Canada.

2. The Friends of Garrett Cumming Research & Muscular Dystrophy Canada HM Toupin Neurological Science Research Chair, Edmonton, 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.2021.0009

Reference39 articles.

1. Fibrodysplasia Ossificans Progressiva: Clinical and Genetic Aspects

2. A recurrent mutation in the BMP type I receptor ACVR1 causes inherited and sporadic fibrodysplasia ossificans progressiva

3. Fibrodysplasia Ossificans Progressiva: Clinical Course, Genetic Mutations and Genotype-Phenotype Correlation

4. ACVR1 R206H receptor mutation causes fibrodysplasia ossificans progressiva by imparting responsiveness to activin A

5. Neofunction of ACVR1 in fibrodysplasia ossificans progressiva

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1. Oxidative phosphorylation is a pivotal therapeutic target of fibrodysplasia ossificans progressiva;Life Science Alliance;2024-02-16

2. Lysosomal degradation of ACVR1-Activin complexes negatively regulates signaling of Activins and Bone Morphogenetic Proteins;2024-01-31

3. Navigating the Complex Landscape of Fibrodysplasia Ossificans Progressiva: From Current Paradigms to Therapeutic Frontiers;Genes;2023-11-30

4. Antiviral Efficacy of RNase H-Dependent Gapmer Antisense Oligonucleotides against Japanese Encephalitis Virus;International Journal of Molecular Sciences;2023-10-02

5. Multi-omics therapeutic perspective on ACVR1 gene: from genetic alterations to potential targeting;Briefings in Functional Genomics;2022-08-25