Genetically Modified Stem Cells May Have Therapeutic Application to X-SCID
Date: 3.6.2007
A study demonstrating therapeutic utilization of human stem cells, including potential application to X-linked Severe Combined Immunodeficiency (XSCID or “Bubble-Boy” disease) was presented today at the 10th Annual Meeting of the American Society of Gene Therapy (ASGT) in Seattle.
X-linked Severe Combined Immunodeficiency (XSCID) is a rare genetic disease caused by defects in an essential immune hormone receptor called the common gamma chain (γc), found on the surface of lymphocytes and other immune blood cells. Patients with XCID have immune cells that lack this receptor and both fail to develop properly, as well as do not function normally to protect patients against infections.
A team of scientists led by Dr. Luigi Naldini from the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), Milan and Sangamo BioSciences Inc., Richmond, CA, are developing a novel approach to genetically modify embryonic and tissue stem cells for therapeutic use. This approach can potentially be applied to many diseases, including X-SCID.
Using technology developed by Sangamo, the scientists showed gene correction or precise addition of a gene in human stem cells at unprecedented levels.
“This is a significant advance for the potential therapeutic use of stem cells,” says Dr. Angelo Lombardo, lead author of the study. “Stem cells are the body’s natural resource for regeneration and repair and the ability to efficiently insert a therapeutic gene in a predetermined location or to correct a mutated gene in a patient’s stem cells may enable us to provide a long term solution for many genetic diseases. The powerful combination of Sangamo’s ZFN™ technology and our highly efficient viral delivery platform was the key factor for obtaining unprecedented gene modification efficiency in these therapeutically important cells”.
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