Gene therapy’s time seems to have come

July 14, 2013


Two boys born with a fatally flawed gene that leaves them defenseless against common infections have returned to their homes in Argentina and Chile after receiving an experimental gene therapy at Boston Children’s Hospital.

Untreated, babies with their “bubble boy” disease would die in infancy, but in 2010 and 2012, doctors inserted good copies of the gene into their bone marrow cells, restoring the boys’ immune systems. Both are now well into toddlerhood.

Years after it was hyped, then vilified after the death of a teenager, then mostly forgotten by the general public, gene therapy has rebounded, and hospitals, companies, and investors in the Boston area have jumped on the bandwagon. Patients are enrolling in a growing number of clinical trials here, and in some cases showing dramatic improvement. The technique, in which doctors “infect” patients’ cells with viruses engineered to carry useful genes, has matured and evolved.

With a handful of experimental trials open or in the works at local hospitals, gene therapy seems to have found its footing. Dr. David Williams, director of translational research at Boston Children’s Hospital, said he plans this summer to bring together key players from area hospitals to share the expertise Children’s has gained from launching trials. The trials include one for a rare, genetic immune system disease called Wiskott-Aldrich syndrome and another scheduled to begin this summer for the neurodegenerative disease adrenoleukodystrophy, best known from the role it played in the movie “Lorenzo’s Oil.”

This story was originally published by The Boston Globe. Read the whole story here.  

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