CRISPR-Generated Animal Models of Duchenne Muscular Dystrophy.

Genes
Kenji Rowel Q LimToshifumi Yokota

Abstract

Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive neuromuscular disorder most commonly caused by mutations disrupting the reading frame of the dystrophin (DMD) gene. DMD codes for dystrophin, which is critical for maintaining the integrity of muscle cell membranes. Without dystrophin, muscle cells receive heightened mechanical stress, becoming more susceptible to damage. An active body of research continues to explore therapeutic treatments for DMD as well as to further our understanding of the disease. These efforts rely on having reliable animal models that accurately recapitulate disease presentation in humans. While current animal models of DMD have served this purpose well to some extent, each has its own limitations. To help overcome this, clustered regularly interspaced short palindromic repeat (CRISPR)-based technology has been extremely useful in creating novel animal models for DMD. This review focuses on animal models developed for DMD that have been created using CRISPR, their advantages and disadvantages as well as their applications in the DMD field.

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Citations

Sep 20, 2020·Journal of Personalized Medicine·Rika Maruyama, Toshifumi Yokota
Feb 12, 2021·Genes·Virginia Arechavala-Gomeza, Lidia Gonzalez-Quereda
Jun 22, 2021·Frontiers in Pharmacology·Leonela LuceFlorencia Giliberto
Jul 17, 2021·Cell & Bioscience·Xiaodong ZouXiaochun Tang
Jul 14, 2021·Cardiovascular Research·Francesco CanonicoDomenico D'Amario
Aug 20, 2021·Transgenic Research·M I ZaynitdinovaS A Smirnikhina

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Methods Mentioned

BETA
targeted mutations
transgenic

Software Mentioned

TALEN

Related Concepts

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Clustered regularly interspaced short palindromic repeats (CRISPR) are DNA sequences in the genome that are recognized and cleaved by CRISPR-associated proteins (Cas). CRISPR-Cas system enables the editing of genes to create or correct mutations. Discover the latest research on CRISPR here.

CRISPR for Genome Editing

Genome editing technologies enable the editing of genes to create or correct mutations. Clustered regularly interspaced short palindromic repeats (CRISPR) are DNA sequences in the genome that are recognized and cleaved by CRISPR-associated proteins (Cas). Here is the latest research on the use of CRISPR-Cas system in gene editing.

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