MEMPHIS, Tenn. – Researchers from St. Jude Children’s Research Hospital have cured babies with “bubble boy” disease through gene therapy involving a re-engineered virus, according to a newly published study.
St. Jude and UCSF Benioff Children’s Hospital San Francisco treated the children enrolled in the clinical trial with gene therapy developed by St. Jude’s Brian Sorrentino, the study’s senior author, who led groundbreaking gene therapy research before his death in November at 60 years old. Study co-author Stephen Gottschalk, chair of the St. Jude Department of Bone Marrow Transplantation and Cellular Therapy, said the researchers hope the therapy will be a template for treating other blood disorders.
How the therapy worksThe gene therapy works like this: A deactivated virus is inserted into the patient’s bone marrow, which delivers the correct gene copy into blood stem cells, replacing the defective one. These cells are then frozen and undergo testing. The proper immune cells were found within three months of the treatment in all but one patient, who needed a second dose of gene therapy, St. Jude says.
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