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Scientists at St. Jude Children’s Research Hospital have pinpointed in DNA the gene therapy for safe cures of infants with SCID-X1

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“We now have a robust pipeline to monitor the safety of lentiviral gene therapies,” said senior co-corresponding author Jiyang Yu, PhD, St. Jude Department of Computational Biology interim chair. “This really gives hope to patients with genetic diseases that can be cured by lentiviral gene therapy. It looks like we cured bubble boy disease safely in these patients.” The St. Jude team published the entire computational pipeline together with the manuscript.

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