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Related Concept Videos

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...
Myasthenia Gravis: Overview and Treatment01:20

Myasthenia Gravis: Overview and Treatment

Myasthenia gravis is a neuromuscular transmission disorder characterized by weakness and increased fatigability of skeletal muscles. It is an autoimmune disease affecting approximately one in 2000 people, where antibodies against the α1 subunit of nicotinic acetylcholine receptors are produced.
These antibodies interfere with the function of the nicotinic receptors in three ways: by binding to the receptor and disrupting acetylcholine binding; by causing cross-linking of receptors which leads...
Disorders of the Skeletal Muscle01:28

Disorders of the Skeletal Muscle

The clinical conditions affecting the skeletal muscle tissue are broadly categorized as musculoskeletal and neuromuscular disorders.
Musculoskeletal disorders
Musculoskeletal disorders involve injuries and conditions affecting the skeletal muscles and associated connective tissues. These disorders can arise from acute biomechanical stresses or chronic overuse and can occur across different age groups. Common injuries include sprains, fractures, and muscular strains, often resulting from...
Classification of Skeletal Muscle Relaxants01:28

Classification of Skeletal Muscle Relaxants

Skeletal muscle relaxants are a group of drugs that can reduce muscle stiffness and induce temporary paralysis to relieve pain. These agents can act centrally to reduce muscle tone or spasms in painful conditions such as multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), or spinal injuries; they are called antispasmodics or spasmolytics.
Peripherally acting skeletal muscle relaxants interfere with the neurotransmission at the neuromuscular end plate to induce paralysis during...
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...
Myasthenia Gravis: Diagnostic Tests01:15

Myasthenia Gravis: Diagnostic Tests

Myasthenia gravis is an autoimmune condition affecting neuromuscular transmission, causing generalized weakness in skeletal muscles. Initial diagnoses rely on patients' signs, symptoms, and medical history. The challenge lies in distinguishing myasthenia from other muscular dystrophies. An important diagnostic feature is the significant improvement of symptoms after administering anticholinesterase inhibitors.
The edrophonium test is a diagnostic tool for myasthenia gravis. It involves...

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Related Experiment Video

Updated: Jun 23, 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

Therapy for neuromuscular disorders.

Andrea L H Arnett1, Joel R Chamberlain, Jeffrey S Chamberlain

  • 1Medical Scientist Training Program, University of Washington, Seattle, WA 98195-7720, United States. alarnett@u.washington.edu

Current Opinion in Genetics & Development
|May 5, 2009
PubMed
Summary
This summary is machine-generated.

Gene therapy, including antisense exon skipping and RNA interference, shows promise for treating neuromuscular disorders. Advances in viral vectors and stem cell therapy offer new hope for effective treatments.

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Last Updated: Jun 23, 2026

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

  • Neuromuscular Disorders
  • Gene Therapy
  • Stem Cell Therapy

Background:

  • Significant progress has been made in developing therapeutic strategies for both recessive and dominant forms of neuromuscular disorders.
  • Antisense-mediated exon skipping is a key gene therapy approach being investigated to correct mutations in the dystrophin gene.
  • Emerging stem cell therapies utilize newly discovered multipotent cell lineages for muscle engraftment.

Purpose of the Study:

  • To review recent advancements in therapeutic approaches for neuromuscular disorders.
  • To highlight the potential of gene therapy techniques like exon skipping and RNA interference.
  • To discuss the prospects of stem cell therapy in treating muscle diseases.

Main Methods:

  • Antisense oligonucleotide therapy and RNA interference for dominant genetic muscle diseases.
  • Development and refinement of adeno-associated viral vectors for gene delivery.
  • Exploration of multipotent cell lineages for stem cell-based therapies.

Main Results:

  • Successful restoration of dystrophin expression in animal models using exon skipping.
  • Development of strategies targeting mutant gene product elimination for dominant disorders.
  • Identification of stem cell populations capable of muscle engraftment via vascular delivery.

Conclusions:

  • Gene therapy and stem cell therapy show considerable promise for treating a variety of neuromuscular disorders.
  • Ongoing research and refinement of delivery vectors are paving the way for clinical trials.
  • These advancements represent substantial progress toward effective therapies for debilitating muscle diseases.