Researchers at UCSF are seeing encouraging early results in a clinical trial involving young kids with a rare condition known as "bubble baby disease."
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Doctors are using gene therapy to treat children with Severe Combined Immunodeficiency – or SCID. They often have to live in a bubble because the slightest infection could kill them. The standard treatment is a bone marrow transplant, but that has its issues – including the risk of rejection.
"To overcome some of these problems, in particular a rejection, we often give high doses of chemotherapy," Dr. Mort Cowan, UCSF Pediatrics Professor, told KCBS Radio's "As Prescribed" on Thursday.
He and fellow pediatrician, Dr. Jennifer Puck, have been testing a new technique on patients with one type of SCID.
They take the patient's own cells and correct the mutation.
"We do that actually outside of the patient's body in the laboratory, and when the cells are corrected we reinfuse them into the patient and, of course, the patient doesn't see them as foreign," Puck explained. "They are the patient's own cells, so they are accepted."
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