Author

E. Montini

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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. · New England Journal of Medic... · 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. · Molecular Therapy · 0 citations

#gene editing Open access Sep 2026

Targeting white matter astrocytes with a Gfap-disruptive Cas9 nuclease benefits a murine model of Alexander disease.

Astrocytes are intriguing therapeutic targets due to their crucial role in initiating and promoting demyelination in the central nervous system. By efficient on-target editing, we can permanently disrupt detrimental genes and restore normal physiological processes. Here, we have optimized an AAV platform to deliver the...

Anne E. J. Hillen, I. Capuano, Barbara Gega et al. · Molecular Therapy · 0 citations