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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...
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...
Satellite Stem Cells and Muscular Dystrophy01:21

Satellite Stem Cells and Muscular Dystrophy

Satellite stem cells or myosatellite cells are quiescent stem cells that Alexander Mauro first identified in 1961. These cells are located between the sarcolemma, the plasma membrane of muscle fibers, and the basal lamina, the connective tissue sheath covering it. These mononucleated cells are activated in response to muscle injury, can transform into myoblasts, and may form or repair muscle fibers. Myosatellite cells can provide additional myonuclei for muscle regeneration or return to a...
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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...
Myasthenia Gravis ll: Pathophysiology01:22

Myasthenia Gravis ll: Pathophysiology

The disease process of myasthenia gravis begins at the neuromuscular junction, where antibodies attack key proteins needed for muscle activation. This immune reaction weakens signal transmission, leading to the characteristic muscle fatigue and weakness that define the condition.Immune-Mediated DamageIn most individuals, antibodies target acetylcholine receptors (AChRs) on the postsynaptic membrane of muscle cells. By blocking acetylcholine binding, these antibodies prevent the nerve signal...

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Related Experiment Video

Updated: Jun 22, 2026

Purification and Transplantation of Myogenic Progenitor Cell Derived Exosomes to Improve Cardiac Function in Duchenne Muscular Dystrophic Mice
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Purification and Transplantation of Myogenic Progenitor Cell Derived Exosomes to Improve Cardiac Function in Duchenne Muscular Dystrophic Mice

Published on: April 10, 2019

Gene therapy for muscular dystrophy: current progress and future prospects.

Capucine Trollet1, Takis Athanasopoulos, Linda Popplewell

  • 1School of Biological Sciences, Royal Holloway-University of London, Egham, TW20 0EX, Surrey, UK.

Expert Opinion on Biological Therapy
|June 17, 2009
PubMed
Summary

Gene therapies offer new hope for muscular dystrophies, particularly Duchenne muscular dystrophy (DMD). Advances in exon skipping and gene replacement strategies show promise in clinical trials for treating this progressive muscle-wasting disease.

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Purification and Transplantation of Myogenic Progenitor Cell Derived Exosomes to Improve Cardiac Function in Duchenne Muscular Dystrophic Mice
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Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders
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Published on: April 3, 2021

Area of Science:

  • Genetics
  • Molecular Biology
  • Neurology

Background:

  • Muscular dystrophies are inherited disorders causing progressive muscle weakness and degeneration.
  • Current treatments for muscular dystrophies, especially Duchenne muscular dystrophy (DMD), are inadequate.
  • Duchenne muscular dystrophy is the most common and severe form of muscular dystrophy.

Purpose of the Study:

  • To review advances in gene-based therapies for muscular dystrophies.
  • To highlight the potential of novel therapeutic strategies for Duchenne muscular dystrophy.
  • To discuss the progress of clinical trials for genetic and cell-based treatments.

Main Methods:

  • Review of current gene-based therapeutic strategies including gene replacement, RNA-based approaches, and cell-based gene therapy.
  • Focus on exon skipping induced by antisense oligonucleotides.
  • Exploration of gene delivery systems like adeno-associated virus (AAV) and lentivirus vectors.

Main Results:

  • Exon skipping has demonstrated proof-of-concept in animal models and clinical trials, offering a promising therapy for a subset of DMD patients.
  • Gene delivery strategies using AAV vectors enable efficient in vivo gene transfer to skeletal muscles.
  • Lentivirus vectors show potential for ex vivo gene modification combined with cell-based therapies.

Conclusions:

  • Gene-based therapies, including exon skipping and gene replacement, hold significant promise for treating Duchenne muscular dystrophy.
  • Advancements in gene delivery vectors are crucial for effective in vivo and ex vivo therapeutic strategies.
  • Ongoing clinical trials indicate a hopeful future for patients with muscular dystrophies through genetic interventions.