Article

Etuvetidigene autotemcel for the treatment of Wiskott-Aldrich Syndrome

2025-12-04

Abstract excerpt

<h4>ABSTRACT</h4> <h4>BACKGROUND:</h4> Wiskott-Aldrich Syndrome (WAS) is a rare, X-linked, life-threatening inborn error of immunity and platelet disorder caused by WAS protein (WASP)-encoding gene mutations. Etuvetidigene autotemcel (etu-cel) is an autologous gene therapy (GT) consisting of hematopoietic stem progenitor cell (HSPCs) transduced ex vivo with a lentiviral vector encoding human WAS cDNA. <h4>METH...

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Literature Corpus work: e6f76e52-1638-5866-8f5d-b3f2ac724b2c
DOI: 10.64898/2025.11.25.25340584

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