Forty Seven, Inc. has developed an antibody therapy to block a protein called CD47 that is found on the surface of cancer cells. CD47 acts as a 'don't eat me'…
A team at UC Davis is taking a patient's blood forming stem cells and inserting three anti-HIV genes into them and then returning them to the individual to help rebuild…
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…
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…
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…
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…
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…
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…
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…
Up to 12,000 Americans suffer a spinal cord injury (SCI) each year. This condition leads to a high level of permanent disability and decreased life expectancy. Currently, there are no…
