Thalassemia Fact Sheet

CIRM funds many projects seeking to better understand thalassemia and to translate those discoveries into new therapies.

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Thalassemias are genetic diseases that most often require inheriting the defective gene from both the mother and father. The form of the disease called Beta thalassemia is most common among people of Mediterranean origin. An estimated 1,000 individuals in the U.S. are living with the disease.

Beta thalassemia is often fatal due to organ damage. But that damage is a two-step process involving the disease itself and the therapy currently used to manage it. The genetic defect causes the patients to produce red blood cells with poorly functioning hemoglobin, the protein that carries oxygen to all our tissues. To correct this, doctors give patients frequent blood transfusions. Those in turn lead to a build up of iron in their blood and if this iron is not properly filtered out, it leads to potentially lethal organ damage.

Bone marrow transplants have been used to treat people with severe forms of Beta thalassemia. The procedure can give the patients a new set of blood-forming stem cells that have the correct gene for hemoglobin, but it is very risky. It currently requires using stem cells from a donor. Such transplants also require extensive pre-treatment, such as undergoing high-dose chemotherapy or radiation, to get rid of the faulty stem cells and risk rejection by the patient’s immune system.

Research funded by California’s stem cell agency is working to develop a safer form of bone marrow transplant for these patients. They are developing a way to genetically alter the patient’s own stem cells so that they can produce red cells with the correct hemoglobin.

CIRM Grants Targeting Blood Diseases Including Thalassemia

Researcher name Institution Grant Title Approved funds
Inder Verma Salk Institute for Biological Studies Development of a cell and gene based therapy for hemophilia $2,298,634
Dianne McKay University of California, San Diego Role of intracytoplasmic pattern recognition receptors in HSC engraftment $615,639
Irving Weissman Stanford University Prospective isolation of hESC-derived hematopoietic and cardiomyocyte stem cells $2,471,386
Tippi MacKenzie University of California, San Francisco In Utero Embryonic Stem Cell Transplantation to Treat Congenital Anomalies $2,661,742
David Davidson Bluebird Bio A Phase 1/2, Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects with β-Thalassemia by Transplantation of Autologous Hematopoietic Stem Cells [REDACTED] $0
Donald Kohn University of California, Los Angeles Beta-Globin Gene Correction of Sickle Cell Disease in Hematopoietic Stem Cells $1,652,076
Luisa Iruela-Arispe University of California, Los Angeles Molecular Characterization and Functional Exploration of Hemogenic Endothelium $1,371,477
Steven Artandi Stanford University Self-renewal and senescence in iPS cells derived from patients with a stem cell disease $931,285
Fyodor Urnov Sangamo BioSciences, Inc. A Treatment For Beta-thalassemia via High-Efficiency Targeted Genome Editing of Hematopoietic Stem Cells $2,760,540
Donald Kohn University of California, Los Angeles Stem Cell Gene Therapy for Sickle Cell Disease $8,833,695
Hiromitsu Nakauchi Stanford University Generation of functional cells and organs from iPSCs $5,427,512
Fred Gage Salk Institute for Biological Studies Development of Induced Pluripotent Stem Cells for Modeling Human Disease $1,737,720
Lili Yang University of California, Los Angeles Differentiation of Human Hematopoietic Stem Cells into iNKT Cells $614,400
Cornelis Murre University of California, San Diego Generation of long-term cultures of human hematopoietic multipotent progenitors from embryonic stem cells $473,952
Hanna Mikkola University of California, Los Angeles Improving microenvironments to promote hematopoietic stem cell development from human embryonic stem cells $550,241
Inder Verma Salk Institute for Biological Studies Curing Hematological Diseases $5,979,252
John Zaia City of Hope The Innovation-Alpha Clinic for Cellular Therapies (I-ACT) – A Program for the Development and Delivery of Innovative Cell-based Treatments and Cures for Life-threatening Diseases. $8,000,000
Nicholas Gascoigne Scripps Research Institute Role of Innate Immunity in hematopoeitic stem cell-mediated allograft tolerance $1,705,554
Hanna Mikkola University of California, Los Angeles A suite of engineered human pluripotent stem cell lines to facilitate the generation of hematopoietic stem cells $1,382,400
Tippi MacKenzie University of California, San Francisco Maternal and Fetal Immune Responses to In Utero Hematopoietic Stem Cell Transplantation $1,230,869
Irving Weissman Stanford University Identification and isolation of transplantable human hematopoietic stem cells from pluripotent cell lines; two steps from primitive hematopoiesis to transplantable definitive cells, and non-toxic conditioning of hosts for hematopoeitic stem cell transplan $1,341,403
David Raulet University of California, Berkeley Inactivating NK cell reactivity to facilitate transplantation of stem cell derived tissue $952,896
Paula Cannon University of Southern California Site-specific gene editing in hematopoietic stem cells as an anti-HIV therapy $1,499,400
Judith Shizuru Stanford University Purified allogeneic hematopoietic stem cells as a platform for tolerance induction $1,233,275
Andrew Leavitt University of California, San Francisco Small molecule tools and scale-up technologies to expand human umbilical cord blood stem and progenitor cells for clinical and research use $1,416,600
Irving Weissman Stanford University Antibody tools to deplete or isolate teratogenic, cardiac, and blood stem cells from hESCs $1,463,814
Matthew Porteus Stanford University Pre-clinical development of gene correction therapy of hematopoietic stem cells for SCID-X1 $874,877
Ann Zovein University of California, San Francisco Human endothelial reprogramming for hematopoietic stem cell therapy. $2,319,784
John Chute University of California, Los Angeles Niche-Focused Research: Discovery & Development of Hematopoietic Regenerative Factors $5,174,715

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