Scientists develop universal donor stem celltherapy to treat degenerative brain diseases in a preclinical study cityofhope advscinews
City of Hope's Yanhong Shi, Ph.D., and her colleagues engineered healthy human skin cells containing the functional aspartoacylase gene into induced pluripotent stem cells and then differentiated the iPSCs into oligodendroglial progenitor cells, the precursor cells that produce myelin, an insulating sheath that wraps around nerve fibers. Like a bullet train, myelin sheaths facilitate the light-speed transport of information along neuronal axons.
Scientists at City of Hope have developed universal donor stem cells that could one day provide lifesaving therapy to children with lethal brain conditions, such as Canavan disease, as well as to people with other degenerative diseases, such as Alzheimer's and multiple sclerosis.
This is the first time stem cells have been engineered to become universal donors for cell therapy targeting diseases of the central nervous system, Shi said. This"off-the-shelf" approach can provide patients who need cell therapy with lifesaving treatments three to six months earlier. Shi and her colleagues engineered healthy human skin cells containing the functional aspartoacylase gene into induced and then differentiated the iPSCs into oligodendroglial progenitor cells, the precursor cells that produce myelin, an insulating sheath that wraps around nerve fibers. Like a bullet train, myelin sheaths facilitate the light-speed transport of information along neuronal axons.
The treated Canavan disease animal models exhibited increased ASPA activity compared to the control mice and had a reduction in the toxic accumulation of the metabolite N-acetyl-L-aspartate in the brain. Too much NAA has been linked to impaired motor function, mental deficiencies and premature death.
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