Cancer is a leading cause of death in California. Many cancers resist current therapies due to therapy-resistant cancer stem cells (CSCs). Scientists at UCSD have discovered a protein, ROR1, that…
This trial proposes to replace SCID patients' dysfunctional immune cells with healthy ones using a safer form of bone marrow transplant (BMT). Current BMT procedures must use toxic chemotherapy to…
Calimmune is genetically modifying patients' own blood-forming stem cells (also known as bone marrow stem cells) so they can produce immune cells—the ones normally destroyed by the HIV virus—that cannot…
In ADA-SCID, allogeneic hematopoietic (blood) stem cell transplants from non-matched sibling donors are a high risk procedure. Additionally, the efficacy of chronic enzyme replacement therapy is uncertain in the long-term. A team at…
St. Jude Children's Research Hospital is teaming up with UC San Francisco to repair the damaged immune system of children born with SCID. They will genetically modify the patient's own…
X-linked Chronic Granulomatous Disease (X-CGD) is a rare immune disorder that prevents white blood cells from killing foreign invaders. This results in severe, recurrent infections that can impact quality and length of…
Sickle cell disease (SCD) is caused by a genetic mutation in the hemoglobin gene which causes red blood cells to "sickle" under conditions of low oxygen. SCD affects 1:500 African-Americans and…
Dr. MacKenzie and her team at UCSF are using hematopoietic stem cells (HSCs) to treat babies in the womb who have alpha thalassemia major, a blood disorder that is almost always fatal. Current…
Retinitis pigmentosa (RP) is a severe form of blindness that runs in families with an incidence of 1:4000. A team at UC Irvine, is using cells called retinal progenitor cells to…
ALS is a devastating neurodegenerative disease with no cure that specifically affect a patient's motor neurons in the brain. A team at Cedars-Sinai is transplanting millions of genetically engineered stem cells…
Get the latest CIRM news, information on upcoming meetings, and funding opportunities delivered to your inbox.
