AIDS virus used in gene therapy to fix ‘bubble baby’ disease - Fox News
May 11, 2021 1 min, 10 secs

A gene therapy that makes use of an unlikely helper, the AIDS virus, gave a working immune system to 48 babies and toddlers who were born without one, doctors reported Tuesday.

Results show that all but two of the 50 children who were given the experimental therapy in a study now have healthy germ-fighting abilities.

Josselyn Kish suffered rashes, painful shingles and frequent diarrhea, said her mother, Kim Carter.

"We’re taking what otherwise would have been a fatal disease" and healing most of these children with a single treatment, said study leader Dr.

The other two children who weren’t helped by the gene therapy later had successful bone marrow transplants.

This October 2013 photo provided by the family shows Josselyn Kish on the day she received a gene therapy treatment at UCLA Mattel Children’s Hospital in Los Angeles. .

As a baby, she suffered rashes, painful shingles and frequent diarrhea, said her mother, Kim Carter.

After the gene therapy, "she was better right away," Carter said.

The fact the treatment seems safe across multiple hospitals performing it makes the study "very powerful," said Dr.

He had no role in the new study but he and his colleagues have performed a similar gene therapy on 17 other children with SCID

Results of the UCLA-led study were published Tuesday by the New England Journal of Medicine and presented at an online American Society of Gene & Cell Therapy conference


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