Antisense-Mediated Skipping of Dysferlin Exons in Control and Dysferlinopathy Patient-Derived Cells-Reference-Cited by-同舟云学术

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Antisense-Mediated Skipping of Dysferlin Exons in Control and Dysferlinopathy Patient-Derived Cells

Published:2020-04-01 Issue:2 Volume:30 Page:71-79
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Verwey Nisha¹,Gazzoli Isabella¹,Krause Sabine²,Mamchaoui Kamel³,Mouly Vincent³,Aartsma-Rus Annemieke¹

Affiliation:

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

2. Department of Neurology, Friedrich-Baur-Institute, Ludwig-Maximilians-University of Munich, Munchen, Germany.

3. Sorbonne Université, INSERM, Institut de Myologie, Myology Research Center, CRM, Paris, France.

Publisher

Mary Ann Liebert Inc

Subject

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

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2019.0788

Reference32 articles.

1. A gene related to Caenorhabditis elegans spermatogenesis factor fer-1 is mutated in limb-girdle muscular dystrophy type 2B

2. Dysferlin, a novel skeletal muscle gene, is mutated in Miyoshi myopathy and limb girdle muscular dystrophy

3. A Systematic Review and Meta-analysis on the Epidemiology of the Muscular Dystrophies

4. Prevalence of Muscular Dystrophies: A Systematic Literature Review

5. Analysis of theDYSFmutational spectrum in a large cohort of patients

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1. Identification of bazedoxifene for the treatment of LGMD R2 by high throughput screening;2024-03-04

2. Current Strategies of Muscular Dystrophy Therapeutics: An Overview;Methods in Molecular Biology;2022-11-19

3. Antisense Oligonucleotide-Mediated Exon-skipping Therapies: Precision Medicine Spreading from Duchenne Muscular Dystrophy;JMA Journal;2021

4. Dual Fluorescence Splicing Reporter Minigene Identifies an Antisense Oligonucleotide to Skip Exon v8 of the CD44 Gene;International Journal of Molecular Sciences;2020-11-30

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