Lentiviral Gene Transfer Corrects Immune Abnormalities in XIAP Deficiency.

Joseph TopalNeelam PanchalAmairelys BarroetaAnna RoppeltAnnelotte MuddeH Bobby GasparAdrian J ThrasherBenjamin C HoughtonClaire Booth

Published in: Journal of clinical immunology (2022)

Gene correction of HSCs recovers XIAP-dependent immune defects and could offer a treatment option for patients with XIAP deficiency.

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