Author

Stefan A. Muljo

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#gene editing Open access Oct 2026

MATRIN3 deficiency in human cells triggers an autoinflammatory response via cGAS-STING activation.

MATRIN3 (MATR3) is a broadly expressed nuclear RNA-binding protein. However, the mechanisms by which MATR3 maintains human cellular health remain poorly understood. In this study, we employed gene editing to model MATR3 deficiency in human induced pluripotent stem cells (iPSCs) and the HAP1 haploid cell line. To invest...

Zohirul Islam, A. Polash, Colin L. Sweeney et al. · Proceedings of the National... · 0 citations

Nov 2026

Adult hematopoietic stem cells activate a normally fetal-restricted program to functionally expand ex vivo.

The ability to robustly expand transplantable hematopoietic stem cells (HSCs) ex vivo enables basic science and clinical therapies otherwise hindered by the scarcity of these self-renewing multipotent cells. Despite recent improvements in long-term ex vivo HSC expansion conditions, the molecular mechanisms required for...

Kyomi J. Igarashi, M. Nicholls, Leonid Olender et al. · Blood · 0 citations