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Pre-clinical development of gene correction therapy of hematopoietic stem cells for SCID-X1

  • Post author:
  • Post published:December 20, 2025
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Severe combined immunodeficiency caused by mutations in the IL2RG gene on the x-chromosome (SCID-X1 or "bubble boy disease") is a devastating genetic disease that results in boys not being able…

Continue ReadingPre-clinical development of gene correction therapy of hematopoietic stem cells for SCID-X1

Generation of a functional thymus to induce immune tolerance to stem cell derivatives

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  • Post published:December 20, 2025
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Stem cell research offers the promise of replacing missing or damaged tissues in the treatment of disease. Stem-cell-derived transplants still face problems with rejection as in traditional organ transplants. Several…

Continue ReadingGeneration of a functional thymus to induce immune tolerance to stem cell derivatives

A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants

  • Post author:
  • Post published:December 20, 2025
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Successful stem cell therapy requires the replacement of diseased or dysfunctional stem cells with healthy ones. These healthy stem cells can come from either a donor or can be stem…

Continue ReadingA monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants

Gene Correction of Autologous Hematopoietic Stem Cells in Artemis Deficient SCID

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  • Post published:December 20, 2025
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Artemis is a chemical in all cells in the body that is essential for the normal development of the immune system and repairing damaged DNA. Artemis deficiency (AD) causes Severe…

Continue ReadingGene Correction of Autologous Hematopoietic Stem Cells in Artemis Deficient SCID

A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants

  • Post author:
  • Post published:December 20, 2025
  • Post category:

Successful stem cell therapy requires replacement of diseased or dysfunctional stem cells with healthy ones. These healthy stem cells can come from either a donor or can be stem cells…

Continue ReadingA monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants

Development of RNA-based approaches to stem cell gene therapy for HIV

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  • Post published:December 20, 2025
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Despite significant advances in treatment and prevention programs, HIV infection with progression to Acquired Immunodeficiency Syndrome (AIDS) is still prevalent in California. The CDC Estimates >56,000 new cases of HIV…

Continue ReadingDevelopment of RNA-based approaches to stem cell gene therapy for HIV

Curing Hematological Diseases

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  • Post published:December 20, 2025
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The primary aim of this project is to develop treatments for incurable diseases of the blood and immune system. X-linked Severe Combined Immunodeficiency (X-SCID) and Fanconi anemia (FA) are two…

Continue ReadingCuring Hematological Diseases

Evaluation of Gene Therapy Approaches for Autosomal Recessive Hyper IgE Syndrome Due to Mutations in DOCK8

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  • Post published:September 12, 2024
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Continue ReadingEvaluation of Gene Therapy Approaches for Autosomal Recessive Hyper IgE Syndrome Due to Mutations in DOCK8

Stem Cell Cure for “Bubble Baby” Disease (SCID), Pioneered by UCLA’s Don Kohn

  • Post author:cirmadmin
  • Post published:November 18, 2014
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On November 18th, 2014, a UCLA research team led by Donald Kohn, M.D., announced a breakthrough gene therapy and stem cell cure for "bubble baby" disease, or severe combined immunodefiency…

Continue ReadingStem Cell Cure for “Bubble Baby” Disease (SCID), Pioneered by UCLA’s Don Kohn

Blood cells from Embryonic Stem Cells: Cornelis Murre – CIRM Science Writer’s Seminar

  • Post author:cirmadmin
  • Post published:September 17, 2008
  • Post category:

Cornelis Murre, Ph.D., spoke at the Scientific Writer's Seminar, a workshop presented on September 17, 2008 at CIRM headquarters in San Francisco. Murre has a CIRM grant to develop an…

Continue ReadingBlood cells from Embryonic Stem Cells: Cornelis Murre – CIRM Science Writer’s Seminar
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