St. Jude Children’s Research Hospital and the National Institute of Allergy and Infectious Diseases have developed a gene therapy that rebuilds ravaged immune systems, scientists reported in April.(Photo: Nikki Boertman, The (Memphis) Commercial Appeal)
Children suffering from
the so-called "Bubble Boy" disease could benefit from a new type of<span style="color: Red;">*</span>
gene therapy<span style="color: Red;">*</span>— developed with help from St. Jude Children's Research Hospital — that rebuilds ravaged immune systems, scientists reported Wednesday.
In a study published in the journal
Science Translational Medicine, some of the patients afflicted with X-linked severe combined immunodeficiency disease began producing more immune cells after a correct copy of a gene was introduced via a re-engineered virus to replace a mutant gene in the blood-producing stem cells.
The study offers hope for sufferers of the rare inherited disorder made famous by Texas youngster David Vetter — dubbed "David, the bubble boy" by the media because he lived in a sterile chamber to avoid the infections and pathogens to which he was so vulnerable. He died in 1984 at age 12.
The disorder stems from a mutation of a protein-coding gene that affects males and occurs at a frequency of 1 in every 50,000 to 100,000 live births. It leaves patients with virtually no immune protection.
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The study shows that gene therapy, combined with treatment involving the cancer drug busulfan, can "rebuild the immune system and lead to broad immunity in young adults with this devastating disorder," said Dr. Brian Sorrentino, a member of St. Jude's department of hematology, who was co-senior author of the study.
The study involved five males aged 7 to 23 years. They all suffered from chronic viral infections and were being treated at the National Institute of Allergy and Infectious Diseases, which also helped develop the novel gene therapy.
With the corrected gene and the busulfan, stem cells of the patients became restored and functional, researchers said. About two years after the gene therapy, the first two patients in the trial were producing more T, B and other immune cells. Their overall health also improved, as they put on weight, absorbed more protein and got rid of their infections.
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