Next Article in Journal
Mild Hypophagia and Associated Changes in Feeding-Related Gene Expression and c-Fos Immunoreactivity in Adult Male Rats with Sodium Valproate-Induced Autism
Next Article in Special Issue
A National French Consensus on Gene List for the Diagnosis of Charcot–Marie–Tooth Disease and Related Disorders Using Next-Generation Sequencing
Previous Article in Journal
Initiator-Directed Transcription: Fission Yeast Nmtl Initiator Directs Preinitiation Complex Formation and Transcriptional Initiation
 
 
Review

Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot

Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2R3, Canada
*
Author to whom correspondence should be addressed.
Academic Editors: Marc Bartoli and Svetlana Gorokhova
Genes 2022, 13(2), 257; https://doi.org/10.3390/genes13020257
Received: 28 December 2021 / Revised: 18 January 2022 / Accepted: 19 January 2022 / Published: 28 January 2022
(This article belongs to the Special Issue Genetics of Muscular Disorders)
Duchenne muscular dystrophy (DMD) is a fatal genetic disease affecting children that is caused by a mutation in the gene encoding for dystrophin. In the absence of functional dystrophin, patients experience progressive muscle deterioration, leaving them wheelchair-bound by age 12 and with few patients surviving beyond their third decade of life as the disease advances and causes cardiac and respiratory difficulties. In recent years, an increasing number of antisense and gene therapies have been studied for the treatment of muscular dystrophy; however, few of these therapies focus on treating mutations arising in the N-terminal encoding region of the dystrophin gene. This review summarizes the current state of development of N-terminal antisense and gene therapies for DMD, mainly focusing on exon-skipping therapy for duplications and deletions, as well as microdystrophin therapy. View Full-Text
Keywords: Duchenne muscular dystrophy; exon skipping therapy; antisense oligonucleotide; microdystrophin; adeno-associated virus Duchenne muscular dystrophy; exon skipping therapy; antisense oligonucleotide; microdystrophin; adeno-associated virus
Show Figures

Figure 1

MDPI and ACS Style

Wilton-Clark, H.; Yokota, T. Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot. Genes 2022, 13, 257. https://doi.org/10.3390/genes13020257

AMA Style

Wilton-Clark H, Yokota T. Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot. Genes. 2022; 13(2):257. https://doi.org/10.3390/genes13020257

Chicago/Turabian Style

Wilton-Clark, Harry, and Toshifumi Yokota. 2022. "Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot" Genes 13, no. 2: 257. https://doi.org/10.3390/genes13020257

Find Other Styles
Note that from the first issue of 2016, MDPI journals use article numbers instead of page numbers. See further details here.

Article Access Map by Country/Region

1
Back to TopTop