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Author

L. Naldini

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Clinical trial Sep 2026

Etuvetidigene Autotemcel for the Treatment of Wiskott-Aldrich Syndrome.

BACKGROUND Wiskott-Aldrich syndrome is a rare, X-linked, life-threatening inborn error of immunity and platelet disorder caused by variants in the gene WAS. Etuvetidigene autotemcel (etu-cel) is an autologous gene therapy consisting of hematopoietic stem and progenitor cells that have been transduced ex vivo with a len...

F. Ferrua, S. Cenciarelli, S. Giannelli et al. · 0 citations
#gene editing Open access Sep 2026

p38 MAPK inhibition safeguards genome integrity during gene editing of hematopoietic stem and progenitor cells.

Ex vivo genome editing of hematopoietic stem and progenitor cells (HSPCs) holds significant therapeutic potential but remains constrained by genotoxic risks associated with nuclease-induced DNA double-strand breaks, DNA donor template delivery and sensing, and proliferation-induced stress during ex vivo manipulation. T...

Roberta Vacca, Lucrezia della Volpe, Nicolò Gualandi et al. · 0 citations
#gene editing Review Open access Aug 2026

Unlocking the Curative Potential of Gene Transfer and Editing for Hematopoietic Disorders.

Recent developments and refinements in gene transfer and editing technologies for HSPCs are reviewed, while also discussing the critical limitations and hurdles to clinical translation, as recently presented at the New Investigator Committee Gene Therapy webinar.

Kohei Shiroshita, A. Stolz, C. Malouf et al. · 0 citations

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