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Gene Therapy For Bubble Baby Syndrome May Lead To Blood Cancer

remarked.

Prof Adrian Thrasher has treated eight boys suffering from this life-threatening disorder with the normal gene at the Great Ormond Street Hospital. Additionally, he has treated two young victims afflicted with Chronic Granulomatous Disorder (CGD) using gene transplant, both of whom managed to clear infections that had not responded to treatment previously.

However it is not known about the long-term side effects associated with form of treatment. Concerns of the scientific community on one side, nothing seems to deter the hope of patients like Mark, a 6 year old CGD sufferer, who feel that gene therapy offers a ray of endless hope at the end of the tunnel.

Clearly, more studies are indicated to accurately assess the risk and benefit ratio associated with gene therapy before it can be commercialized.


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