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Gene Therapy Achieves 95% Success in Immune Restoration

10/17/2025

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Summary

Story summary

ADA-SCID gene therapy restored immune function in 59 of 62 children treated from 2012 to 2019, a 95% success rate.
The study involved researchers from the University of California, Los Angeles (UCLA), University College London, and Great Ormond Street Hospital.
The study was published in the New England Journal of Medicine and reported no serious complications and a 100% survival rate.
FDA approval could come within two to three years.

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Story summary

ADA-SCID gene therapy restored immune function in 59 of 62 children treated from 2012 to 2019, a 95% success rate.
The study involved researchers from the University of California, Los Angeles (UCLA), University College London, and Great Ormond Street Hospital.
The study was published in the New England Journal of Medicine and reported no serious complications and a 100% survival rate.
FDA approval could come within two to three years.