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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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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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Stem Cell Therapy for Tissue Regeneration01:21

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Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
Types of Stem Cells used in Stem Cell Therapy
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Regulation of Hematopoietic Stem Cells01:01

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All blood and immune cells are produced from the multipotent hematopoietic stem cells (HSCs) by the process of hematopoiesis. However, they all have a limited life span. In addition, many are depleted in immune surveillance or combatting an injury or infection. This makes blood one of the most regenerative tissues. Hematopoiesis helps replenish these blood and immune cells, restoring the body's normal functioning. However, overproduction of blood and immune cells can make them cancerous or...
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Multipotency of Hematopoietic Stem Cells

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The hematopoietic stem cells or HSCs are multipotent, meaning they can differentiate and give rise to all blood and immune cells. HSCs are maintained in the quiescent stage until an external stimulus initiates their differentiation. The multipotent HSCs exist as two heterogeneous populations, long-term repopulating cells (LTRC) and short-term repopulating cells (STRC). The two HSC populations have different surface markers or receptors and are classified based on quiescence and long-term...
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iPS Cell Differentiation

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The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
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CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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Treating Immunodeficiency through HSC Gene Therapy.

Claire Booth1, H Bobby Gaspar1, Adrian J Thrasher1

  • 1Molecular and Cellular Immunology Section, UCL Institute of Child Health, London, UK; Department of Paediatric Immunology, Great Ormond Street Hospital, London, UK.

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Haematopoietic stem cell gene therapy offers a viable treatment for inherited immune deficiencies. Newer, safer vectors and gene editing technologies like CRISPR/Cas9 promise improved efficacy and biosafety for future therapies.

Keywords:
gene therapylentiviral vectorsprimary immunodeficiency

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Area of Science:

  • Immunology
  • Genetics
  • Biotechnology

Background:

  • Haematopoietic stem cell (HSC) gene therapy has been a successful treatment for inherited immune deficiencies, such as severe combined immune deficiencies (SCID), for 20 years.
  • Early gene therapy trials utilized gamma-retroviral vectors, which, while effective, carried a risk of leukemogenesis.
  • Recent advancements have led to the development of safer vectors with comparable efficacy and improved biosafety profiles.

Purpose of the Study:

  • To review the evolution of gene therapy for inherited immune deficiencies.
  • To highlight the advancements in vector technology and gene editing.
  • To discuss the future potential of these technologies in improving patient outcomes.

Main Methods:

  • Review of clinical trial data and preclinical studies on HSC gene therapy.
  • Analysis of vector safety and efficacy profiles.
  • Examination of emerging gene editing technologies (ZFNs, TALENs, CRISPR/Cas9).

Main Results:

  • First-generation gamma-retroviral vectors showed clinical benefit but posed risks of leukemogenesis.
  • Newer generation vectors demonstrate comparable efficacy with enhanced biosafety.
  • Gene editing platforms offer precise DNA correction, potentially increasing safety and effectiveness.

Conclusions:

  • HSC gene therapy is a proven therapeutic strategy for SCID and other immune disorders.
  • Ongoing development of safer vectors and gene editing technologies is crucial for advancing the field.
  • Targeted gene editing holds significant promise for future gene therapy applications, improving safety and efficacy.