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Autoimmune Disorders01:29

Autoimmune Disorders

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Autoimmune diseases are a group of disorders in which the body's immune system mistakenly attacks its own cells, tissues, and organs. This results from an overactive immune response against substances and tissues normally present in the body. Let's delve into the concept and mechanism of autoimmune diseases from an immune system point of view, explore different causes and examples of such diseases, and discuss potential solutions.
Concept and Mechanism of Autoimmune Diseases
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Gene Therapy00:59

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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...
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Immunotherapy is a treatment that boosts or manipulates the immune system to fight diseases, including cancer. For instance, by stimulating an immune response through vaccinations against viruses that cause cancers, like hepatitis B virus and human papillomavirus, these diseases can be prevented. Nonetheless, some cancer cells can avoid the immune system due to their rapid mutation and division. The immune response to many cancers involves three phases: elimination, equilibrium, and escape.
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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.
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Development of Stem Cell-derived Antigen-specific Regulatory T Cells Against Autoimmunity
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Gene Therapy for Autoimmune Disease.

Shang-An Shu1, Jinjun Wang1, Mi-Hua Tao2

  • 1Division of Rheumatology/Allergy and Clinical Immunology, School of Medicine, University of California, Davis, CA, 95616, USA.

Clinical Reviews in Allergy & Immunology
|October 4, 2014
PubMed
Summary

Gene therapy offers a promising approach for autoimmune diseases by regulating immune responses and restoring tolerance. This review highlights progress in gene identification and therapeutic applications in models and clinical trials.

Keywords:
Animal modelAutoimmune diseaseClinical trialDelivery vectorsGene therapy

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Area of Science:

  • Immunology
  • Molecular Biology
  • Genetics

Background:

  • Understanding the immunological and molecular basis of autoimmune diseases is crucial.
  • Gene therapy emerges as a promising treatment strategy for autoimmune conditions.

Purpose of the Study:

  • To summarize recent advances in identifying genes linked to autoimmune diseases.
  • To review the application of gene therapy in treating and preventing autoimmune diseases.
  • To discuss the benefits and limitations of current gene therapy strategies.

Main Methods:

  • Review of scientific literature on gene therapy for autoimmune diseases.
  • Analysis of studies in animal models and human clinical trials.
  • Identification of genes implicated in autoimmune disease pathogenesis.

Main Results:

  • Gene therapy aims to modulate proinflammatory cytokines and lymphocyte infiltration.
  • Successful gene delivery and expression are key to therapeutic efficacy.
  • Restoring immune tolerance to autoantigens is the primary objective.

Conclusions:

  • Gene therapy holds significant potential for improving clinical outcomes in autoimmune diseases.
  • Further research is needed to develop novel and effective gene therapy strategies.
  • Continued investigation into gene identification and therapeutic delivery is essential.