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Universal Gene Therapy

A biotech startup in San Francisco is working on a way to transform gene therapy from a risky experiment into a routine treatment. Instead of inserting genes directly into patients, MandalMed researchers plan to transfer them into cultured cells, ensure the genes are working properly, and then implant the cells into patients. The general idea isn’t new, but MandalMed’s particular approach is. Rather than harvesting a patient’s cells, modifying them, and re-implanting them-a time-consuming process carried out to avoid immune rejection-the company hopes to establish collections of “immune silent” cells that would be accepted by any patient’s immune system. MandalMed has begun animal studies with cells that have been modified to treat spinal-cord injury and is testing a number of cell types from a variety of tissues for their ability to evade or suppress the immune system. The researchers’ goal is to tailor the cells for such applications as delivering therapeutic proteins to tumors, replacing proteins absent in genetic diseases including hemophilia, and treating multiple sclerosis, stroke, and Alzheimer’s disease.

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