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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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iPS Cell Differentiation01:22

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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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Multipotency of Hematopoietic Stem Cells01:19

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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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Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells
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Gene therapy outpaces haplo for SCID-X1.

Donald B Kohn1

  • 1UNIVERSITY OF CALIFORNIA, LOS ANGELES.

Blood
|June 6, 2015
PubMed
Summary

Autologous gene therapy for infants with X-linked severe combined immune deficiency (SCID-X1) shows promise. This approach may offer improved outcomes and faster immune recovery compared to haploidentical stem cell transplants.

Area of Science:

  • Pediatric Hematology
  • Immunology
  • Gene Therapy

Background:

  • X-linked severe combined immune deficiency (SCID-X1) is a life-threatening condition requiring timely treatment.
  • Hematopoietic stem cell transplantation (HSCT) is a standard treatment for SCID-X1.
  • Finding suitable donors, especially matched sibling donors, can be challenging.

Purpose of the Study:

  • To evaluate the outcomes of autologous gene therapy/hematopoietic stem cell transplantation (HSCT) in infants with SCID-X1.
  • To compare gene therapy/HSCT with haploidentical (haplo) HSCT in infants lacking a matched sibling donor.
  • To assess the immunological reconstitution following gene therapy/HSCT.

Main Methods:

  • The study involved infants diagnosed with SCID-X1 who lacked a matched sibling donor.

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  • Autologous gene therapy followed by HSCT was performed.
  • Outcomes were compared with those of infants who underwent haploidentical HSCT.
  • Immune reconstitution markers were monitored post-transplant.

    • Main Results:

    • Autologous gene therapy/HSCT demonstrated potentially better outcomes compared to haploidentical HSCT.
    • The gene therapy approach obviates the need for pre- and post-transplant immune suppression.
    • Faster immunological reconstitution was observed in patients treated with gene therapy/HSCT.
    • The risk of graft-versus-host disease (GVHD) is eliminated with autologous transplantation.

    Conclusions:

    • Autologous gene therapy/HSCT is a potentially superior alternative for infants with SCID-X1 lacking a matched sibling donor.
    • This approach simplifies treatment by avoiding immune suppression and GVHD risks.
    • Gene therapy offers a promising path towards faster immune recovery in SCID-X1 patients.