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Gene therapy offers hope for "Bubble Boy" disease: study

Xinhua, April 21, 2016 Adjust font size:

A new gene therapy can safely treat older children and young adults afflicted with a severe inherited immunodeficiency disorder known as "Bubble Boy" disease, a U.S. study said Wednesday.

Until now, gene therapy has only succeeded in infants with X-linked severe combined immune deficiency (SCID-X1), but a small clinical trial showed the new treatment restored normal immune function in some older children and young adults for at least two to three years.

SCID-X1 involves a mutation in the IL2RG gene that affects males and occurs in one of every 50,000 to 100,000 live births, leaving them with little to no immune protection.

The new study, published in the U.S. journal Science Translational Medicine, used a re-engineered lentivirus as a vector to deliver a correct copy of the mutant gene to the blood-producing stem cells of patients.

The treatment, developed by researchers at the St. Jude Children's Research Hospital and the U.S. National Institutes of Health, also used the chemotherapy drug busulfan to help the gene-corrected stem cells become re-established and functional in SCID-X1 patients.

A total of five male patients, aged seven to 23, received the treatment. They all had chronic viral infections and other health problems related to SCID-X1 after one or more bone marrow transplants failed to fully correct their immune function.

After treatment, the patients displayed gene-corrected T, B, and natural killer cells, partially restoring normal protective immune responses.

Clinical symptoms such as chronic norovirus infection and a viral skin condition also markedly improved in some patients.

These benefits persisted for six to nine months after treatment in three younger patients and, notably, for two to three years in two older patients.

There was no indication of possible pre-cancer cell proliferation.

One patient, however, died from pre-existing lung damage more than two years after receiving gene therapy, which highlights the importance of earlier intervention before irreversible organ damage occurs.

"Based on the safety and health benefits for older patients reported in this study, we hope this novel gene therapy will help improve immune functioning and transform the lives of younger patients with this devastating disease," said study author Brian Sorrentino at the St. Jude Children's Research Hospital. Enditem