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Article

Restoration of progranulin by engineered hematopoietic stem cell-derived microglia corrects phenotypes of granulin knockout mice.

Science translational medicine - 7 Jan 2026

Ciervo Yuri, Rigoni Pietro, Bucciarelli Linda, Lombi Martina, Pratesi Federico, Bologna Nadia, Accardo Massimo, Recchi Chiara, Milazzo Rita, Poletti Valentina, Biffi Alessandra

Abstract excerpt

Autologous hematopoietic stem/progenitor cell (HSC)-gene therapy (GT) represents a promising therapeutic option for progranulin (PGRN)-related neurodegenerative diseases due to mutations in the PGRN encoding gene (GRN), such as frontotemporal dementia (FTD) and neuronal ceroid lipofuscinosis 11 (CLN11). These conditions are characterized by a deficiency in PGRN, have no cure, and represent an unmet medical need....

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 41499526
DOI: 10.1126/scitranslmed.adw9930

Genes

GRN

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026.

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