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Related Concept Videos

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Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
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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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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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The process of blood cell formation is called hematopoiesis. Hematopoiesis starts early during development, on the seventh day of embryogenesis. This phase of hematopoiesis is called the primitive wave, wherein the extraembryonic yolk sac allows the production of erythroid cells and endothelial cells from a common precursor called hemangioblast. The erythroid cells provide oxygen to support the growth of the rapidly dividing embryo. Hemangioblasts later develop into hematopoietic stem cells or...
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Bone Marrow Gene Therapy for HIV/AIDS.

Elena Herrera-Carrillo1, Ben Berkhout2

  • 1Laboratory of Experimental Virology, Department of Medical Microbiology, Center for Infection and Immunity Amsterdam (CINIMA), Academic Medical Center, University of Amsterdam, Amsterdam 1105 AZ, The Netherlands. e.herreracarrillo@amc.uva.nl.

Viruses
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Summary

Bone marrow gene therapy offers a durable, lifelong treatment for acquired immunodeficiency syndrome (AIDS) by using hematopoietic stem cells (HSCs) to deliver antiviral genes. This approach contrasts with daily drug regimens for managing human immunodeficiency virus (HIV).

Keywords:
HIV-1RNAiantiviralbone marrowgene therapyhematopoietic stem cell (HSC)lentiviral vectorvirus

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Area of Science:

  • * Hematology
  • * Immunology
  • * Gene Therapy

Background:

  • * Chronic immunological diseases, such as acquired immunodeficiency syndrome (AIDS) caused by human immunodeficiency virus (HIV), require long-term management.
  • * Current treatments for HIV/AIDS often involve daily drug regimens, which can be burdensome for patients.

Purpose of the Study:

  • * To review the potential of bone marrow gene therapy as a durable treatment for HIV/AIDS.
  • * To discuss various antiviral strategies, with an emphasis on RNA-based therapies.
  • * To explore the molecular mechanisms and latest advancements in developing a cure for HIV infection.

Main Methods:

  • * Review of existing literature on bone marrow gene therapy for HIV/AIDS.
  • * Discussion of antiviral strategies targeting the virus or host co-factors.
  • * Analysis of vector systems for gene delivery and preclinical testing models.

Main Results:

  • * Bone marrow gene therapy using hematopoietic stem cells (HSCs) offers long-term expression of therapeutic transgenes.
  • * Clinical trials have demonstrated the safety and proof of principle for gene therapy in treating HIV/AIDS.
  • * Various strategies, including RNA-based therapies, are being explored to target HIV replication.

Conclusions:

  • * Bone marrow gene therapy presents a promising lifelong alternative to daily antiretroviral therapy for HIV/AIDS.
  • * Further research into optimal vector systems, antiviral molecules, and preclinical models is crucial.
  • * Combination gene therapy approaches hold potential for a functional cure for HIV infection.