Gene therapy may offer cure for debilitating ‘bubble boy disease’

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Image: New York Times

Omarion was born with a rare genetic disorder called X-linked severe combined immunodeficiency (SCID), better known as the “bubble boy disease.” Caused by a mutated gene on the X chromosome, and almost always limited to males, a baby born with X-linked SCID, or SCID-X1, lacks a working immune system.

[M]edical scientists at St. Jude Children’s Research Hospital in Memphis, Tennessee, were then developing a bold new procedure. The strategy: introduce a normal copy of the faulty gene, designated IL2RG, into a patient’s own stem cells, which then go on to produce the immune system components needed to fight infection. [His mother Kristin] Simpson enrolled Omarion in the clinical study.

Within four months, some of the babies were able to fight infections on their own. All eight of the initial research subjects left the hospital with a healthy immune system.

[W]ith additional trials and continued monitoring of patients, St. Jude hopes that the therapy will earn Food and Drug Administration approval as a treatment within five years.

Simpson, for her part, is already convinced that the therapy can work wonders: Her son doesn’t live in a bubble or, for that matter, in a hospital.

Read full, original post: These scientist may have found a cure for ‘bubble boy’ disease

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