A new variation of gene therapy raises hopes for a safe and effective long-term treatment for X-linked severe combined immunodeficiency syndrome (SCID-X1), a life-threatening heritable disorder. The research was produced by a collaborative research team from Dana-Farber/Boston Children's Cancer and Blood Disorders Center, along with other institutions participating in an international clinical trial that involved boys from the United States and France. SCID-X1, dubbed bubble boy disease after a patient who lived for 12 years in a sterile bubble, is a rare genetic disorder that hinders the ability of individuals to combat infections. Because the disease is carried in an X-chromosome recessive pattern, the disorder occurs almost only in males. The resulting mutations inactivate a gene called IL-2 receptor gamma (IL2RG), severely weakening immune system functions. Left untreated, individuals who inherit the disorder usually die within a year. Previous gene therapy trials conducted in Europe over a decade ago promised dramatic progress, until a quarter of patients developed leukemia about two to five years following treatment. Scientists found that the previously used vectorthe device for transporting the correct gene in therapyinadvertently activated oncogenes, which can cause cancer. In this new study, the vector in use is a self-inactivating gammaretrovirus, which … Continue reading
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