Article
Direct microglia replacement reveals pathologic and therapeutic contributions of brain macrophages to a monogenic neurological disease.
Immunity - 13 May 2025
Aisenberg William H, O'Brien Carleigh A, Sangster Madison, Yaqoob Fazeela, Zhang Yuanchao, Temsamrit Brian, Thom Searlait, Gosse Luca, Chaluvadi Sai, Elfayomi Bilal, Lee Gavin, Polam Vidhur, Levitt Eli M, Liu Gary, Lombroso Sonia I, Nemec Kelsey M, Clowry Gavin, Nieves Cassaundra, Rawat Priyanka, Church Emily, Martinez Daniel, Shoffler Clarissa, Kancheva Daliya, Petucci Christopher, Taylor Deanne, Kofler Julia, Erskine Daniel, Movahedi Kiavash, Bennett Mariko L, Bennett F Chris
Abstract excerpt
Krabbe disease, also named globoid cell (GC) leukodystrophy (GLD) for its distinct lipid-laden macrophages, is a severe leukodystrophy caused by galactosylceramidase (GALC) mutations. Hematopoietic stem cell transplant (HSCT) ameliorates disease and is associated with central nervous system (CNS) engraftment of GALC+ donor macrophages. Yet, the role of macrophages in GLD pathophysiology and HSCT remains unclear....
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