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Related Concept Videos

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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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.
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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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Stem cell research aims to find ways to use stem cells to regenerate and repair cellular damage. Over time, most adult cells undergo the wear and tear of aging and lose their ability to divide and repair themselves. Stem cells do not display a particular morphology or function. Adult stem cells, which exist as a small subset of cells in most tissues, keep dividing and can differentiate into a number of specialized cells generally formed by that tissue. These cells enable the body to renew and...
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Stem-cell Based Engineered Immunity Against HIV Infection in the Humanized Mouse Model
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Stem-cell-based gene therapy for HIV infection.

Anjie Zhen, Scott Kitchen1

  • 1Department of Medicine, Division of Hematology and Oncology, David Geffen School of Medicine at University of California, Los Angeles, CA 90095, USA. skitchen@ucla.edu.

Viruses
|December 26, 2013
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Summary

Gene therapy using hematopoietic stem cells offers a potential cure for HIV. Strategies include creating HIV-resistant immune systems and boosting immunity to eliminate infected cells.

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Area of Science:

  • Immunology
  • Gene Therapy
  • Hematopoietic Stem Cells

Background:

  • Combined anti-retroviral therapy has limitations for HIV treatment.
  • HIV remains a global health challenge requiring curative strategies.

Purpose of the Study:

  • To review gene therapy approaches for HIV cure using human hematopoietic stem cells.
  • To explore methods for generating HIV-resistant immune systems.
  • To discuss strategies for enhancing anti-HIV immunity.

Main Methods:

  • Review of current research in gene therapy for HIV.
  • Focus on humanized mouse models and stem cell manipulation.
  • Discussion of two primary gene therapy strategies.

Main Results:

  • Gene therapy holds promise for a functional HIV cure.
  • Two main strategies are being investigated: resistance and elimination.
  • Advancements in stem cell technology are crucial.

Conclusions:

  • Hematopoietic stem cell-based gene therapy is a promising avenue for HIV cure.
  • Further research is needed to translate these strategies into effective treatments.
  • Overcoming HIV as a lifelong infection is achievable with innovative therapies.