Scientists at the City of Hope are conducting a Phase 1 clinical trial testing a stem cell-based therapy for adult patients with severe sickle cell disease (SCD) - a chronic, debilitating…
UC San Francisco researchers aim to repair the damaged immune system of children born with severe combined immunodeficiency (SCID), a genetic blood disorder in which even a mild infection can…
Viral infection can lead to fatal complications in patients with weakened immune systems resulting from chemotherapy, bone marrow or cord blood transplant, and other forms of inherited or acquired disorders.…
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…
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…
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