Gene therapy could help cure X-SCID
Immunity of kids restored by gene therapy - interesting science news

Scientists have used gene therapy to restore immunity in infants with X-SCID, which is a life-threatening inherited disorder in which the immune cells do not develop correctly. This makes the infants highly susceptible to infections.

The researchers inserted a normal copy of the IL2RG gene in the normal blood-forming stem cells of these infants. A lentivirus was used to deliver the gene which by itself isn’t infectious to the patient.

Within 3-4 months of the therapy the infants were able to to produce normal number of several immune cells such as T cells, B cells and natural killer cells. This was a small clinical trial and hence will be replicated with a larger sample size in the future.

Read the full story: NIH/National Institute of Allergy and Infectious Diseases
Scientific publication: NEJM


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