Stories about: bubble boy syndrome

New hope for gene therapy

Imagine that instead of drugs or surgery, you could use genes to treat or even  prevent diseases. That’s the promise of gene therapy, an experimental technique where a gene is inserted into a patient’s cells to replace a non-functioning or disease carrying gene.

genetherapy2While gene therapy could potentially cure a variety of diseases, the development of gene therapy strategies against SCID-X1, better known as “bubble boy disease,” has been stalled since 2005, when five boys in Europe with SCID-X1, developed leukemia during clinical trials. Now, a new international trial, sponsored in the United States by Children’s Hospital Boston’s David Williams, MD, director of Translational Research, hopes to revive this potentially life-saving treatment.

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