Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Directly Acting Muscle Relaxants: Dantrolene and Botulinum Toxin01:26

Directly Acting Muscle Relaxants: Dantrolene and Botulinum Toxin

Directly acting muscle relaxants like dantrolene and botulinum toxin (BoNT) have distinct mechanisms and applications. Dantrolene, a hydantoin derivative, acts on the ryanodine receptor (RYR1) in skeletal muscle cells. RYR1 are calcium channels present at the sarcoplasmic reticulum membrane. In response to excitation, they release calcium ions from the sarcoplasmic reticulum to the cytosol. Calcium promotes actin-myosin-mediated contraction of muscles.
The binding of dantrolene to the RYR1...
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
Skeletal Muscle Relaxants: Therapeutic Uses01:31

Skeletal Muscle Relaxants: Therapeutic Uses

Skeletal muscle relaxants are used to relax muscle tone and alleviate painful muscle contractions. However, the choice of skeletal muscle relaxants depends on the duration of the surgical procedure in order to minimize potential side effects. Skeletal muscle relaxants like neuromuscular blocking agents [NMBAs] are commonly employed as adjuvants alongside general anesthetics in clinical settings. NMBAs are also used to maintain controlled ventilation during surgery of the larynx or pharynx as...
Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
Pharmacogenetics of Drug Targets: β₂-Adrenergic Receptors, Apo E, Thymidylate Synthase01:11

Pharmacogenetics of Drug Targets: β₂-Adrenergic Receptors, Apo E, Thymidylate Synthase

Genetic polymorphisms in drug targets have emerged as critical determinants of interindividual variability in drug response and toxicity. Pharmacogenomic investigations increasingly focus on identifying these variations to personalize and optimize therapeutic interventions. A drug target may be a receptor, enzyme, or signaling protein involved in pharmacologic responses or disease-related pathways. While early pharmacogenetic studies focused primarily on drug metabolism, current research...

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

The Multi-System Roles of Dp71 Dystrophin Isoforms in Duchenne Muscular Dystrophy.

Muscles (Basel, Switzerland)·2026
Same author

Guanine base modifications in antisense oligonucleotides mitigate acute central nervous system toxicity.

RSC chemical biology·2026
Same author

Unraveling and controlling late-onset neurotoxicity of antisense oligonucleotides through strategic chemical modifications.

Molecular therapy. Nucleic acids·2025
Same author

Local Non-Coding Regulatory Elements in Muscular Dystrophies.

International journal of molecular sciences·2025
Same author

Ensitrelvir suppresses prolonged olfactory abnormalities derived from SARS-CoV-2 infection in hamsters.

Antiviral research·2025
Same author

Brogidirsen and Exon 44 Skipping for Duchenne Muscular Dystrophy: Advances and Challenges in RNA-Based Therapy.

Genes·2025

Related Experiment Video

Updated: Jun 27, 2026

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
06:51

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

Published on: August 10, 2018

RNA Therapeutics Targeting Skeletal Muscle: Emerging Antisense and Gene-Modifying Strategies.

Takayuki Kuroda1, Toshifumi Yokota1,2

  • 1Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.

Biomolecules
|June 26, 2026
PubMed
Summary

RNA therapeutics offer new treatments for skeletal muscle disorders like Duchenne muscular dystrophy (DMD) by correcting genetic defects. Next-generation strategies focus on improving delivery and safety for broader clinical impact.

Keywords:
Duchenne muscular dystrophyadeno-associated virus vectorantibody–oligonucleotide conjugatesantisense oligonucleotidecell-penetrating peptidesclustered regularly interspaced short palindromic repeats and CRISPR-associated (CRISPR-Cas) systemfacioscapulohumeral muscular dystrophymyotonic dystrophy type 1phosphorodiamidate morpholino oligomersmall interfering RNA

More Related Videos

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
07:02

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

Published on: May 11, 2018

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders
10:28

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders

Published on: April 3, 2021

Related Experiment Videos

Last Updated: Jun 27, 2026

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
06:51

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

Published on: August 10, 2018

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
07:02

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

Published on: May 11, 2018

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders
10:28

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders

Published on: April 3, 2021

Area of Science:

  • Biochemistry
  • Genetics
  • Molecular Biology

Background:

  • RNA-based therapeutics are revolutionizing skeletal muscle disorder treatments.
  • Antisense oligonucleotides (ASOs) like PMOs are FDA-approved for Duchenne muscular dystrophy (DMD), inducing exon skipping to restore dystrophin expression.
  • Other RNA modalities include siRNAs for gene silencing and CRISPR for gene editing.

Purpose of the Study:

  • To review recent advances in RNA-based and gene-modifying strategies for skeletal muscle disorders.
  • To highlight challenges and priorities for clinical translation of these therapies.

Main Methods:

  • Review of current antisense oligonucleotide (ASO) and CRISPR-based gene-modifying technologies.
  • Synthesis of next-generation approaches for improved delivery, stability, and durability.
  • Analysis of practical priorities for clinical translation.

Main Results:

  • Significant progress in RNA therapeutics for DMD, with four FDA-approved ASOs.
  • Delivery to skeletal and cardiac muscle, repeat administration, and safety remain key challenges.
  • Next-generation strategies and CRISPR-based gene editing show promise for enhanced efficacy and one-time correction.

Conclusions:

  • RNA therapeutics hold immense potential for skeletal muscle disorders, but delivery and safety hurdles must be overcome.
  • Future research priorities include enhancing muscle/heart delivery, developing controllable safety mechanisms, and establishing standardized outcome relationships.
  • CRISPR-based strategies offer potential for permanent correction but require careful consideration of delivery, immunogenicity, and long-term safety.