Q & A

Severe Combined Immunodeficiency (SCID), or “Bubble Boy Disease,” is caused by a genetic mutation that severely weakens the immune system. Stem cell approaches involve removing the blood-forming stem cells that eventually become immune cells, then modifying them so that they do not have the mutation. These blood-forming stem cells can then be transplanted back into

Sickle cell disease (SCD) is caused by a genetic mutation that causes the body to produce deformed red blood cells. Stem cell approaches involve taking blood-forming stem cells from the bone marrow—the body’s blood cell factory—then modifying them so that they do not have the mutation. These modified cells can then be transplanted back into

Neural stem cells can be engineered to secrete supportive molecules to the cells that are malfunctioning in ALS. Scientists can use stem cells to protect surviving nerve cells in ALS patients and prevent further degeneration.

Scientists are using stem cells from Parkinson’s disease (PD) patients to see if already-discovered drugs might be useful in treating symptoms of the disease. Another approach involves creating functional brain cells in the lab, with the hope that they might replace the cells that are damaged in PD. It’s possible that one day, cells could

Different kinds of stem cells can be engineered to secrete molecules that are known to reduce some of the symptoms of Huntington’s disease. Scientists are working to see if transplanting these cells can treat animal models of Huntington’s disease. Hopefully, this will progress to clinical trials in patients quickly.

Scientists are turning human embryonic stem cells into special cells called oligodendrocyte progenitor cells that have been shown to support the health of the nerves in spinal cord injury. These cells can then be transplanted into the damaged spinal cord of patients and potentially support the health and restore function to the damaged nerves.