‘It’s A Game Changer’: Scientists Find Success In Using Gene Therapy To Treat ‘Bubble Boy’ Syndrome Without Causing Cancer
The process aimed at helping patients born with a severe immune-system deficiency involves removing some blood cells, using a modified HIV virus to insert a missing gene, and returning the cells through an IV. When doctors first tried it 20 years ago, the treatment had unintended effects on other genes, and some patients later developed leukemia.
The Associated Press:
Doctors Use HIV In Gene Therapy To Fix 'Bubble Boy' Disease
They were born without a working germ-fighting system, every infection a threat to their lives. Now eight babies with "bubble boy disease" have had it fixed by a gene therapy made from one of the immune system's worst enemies — HIV, the virus that causes AIDS. A study out Wednesday details how scientists turned this enemy virus into a savior, altering it so it couldn't cause disease and then using it to deliver a gene the boys lacked. (4/17)
The Washington Post:
Gene Therapy Cures Infants With ‘Bubble Boy’ Immune Disease
“It’s a game changer,” said Jennifer Hemall, a pediatric immunologist at Children’s Hospital of Philadelphia, who was not involved in the study. “For immunologists following this disease, gene therapy has always been out there as the hope of the future. It’s exciting to see this wave of treatments actually becoming a reality.” (Wan, 4/17)
NPR:
'Bubble Boy' Disease Helped By Advances In Gene Therapy
Gene therapy has been used successfully over the past decade. Scientists at St. Jude Children's Research Hospital in Memphis, Tenn., modified the procedure for SCID by giving the infants a short course of chemotherapy before introducing the new gene. This helped the new cells take up permanent residence. The babies developed apparently healthy immune systems, according to the new study. "I am thrilled to see these outstanding results," says Ewelina Mamcarz, a transplant physician and first author on the new paper. (Harris, 4/17)
Bloomberg:
‘Bubble Boys’ Cured In Medical Breakthrough Using Gene Therapy
Nearly two decades ago researchers from France and Italy used a similar approach to successfully treat SCID, though about one-third of the patients eventually developed leukemia, starting about a year after treatment. This time, there is no evidence of leukemia in any of the patients treated, said Stephen Gottschalk, chair of bone marrow transplantation at St. Jude. (Cortez, 4/17)
In other news on gene-editing —
Stat:
In A Mouse Study, CRISPR Edits Lung-Disease Gene In Utero
Companies that hope to treat severe inherited diseases via CRISPR genome editing are already testing the technique in adults, while push-the-envelope types are arguing for repairing defective genes much earlier — in IVF embryos so new they’re still in a lab dish (the “CRISPR babies” route). Now scientists in Philadelphia have taken preliminary steps toward a possible middle way: They injected CRISPR into the amniotic fluid of pregnant mice, editing a lung-disease-causing gene in a small number of mouse fetuses, they reported on Wednesday. (Begley, 4/17)
