Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency

Abstract: Patients with mutations in the UNC13D gene (coding for Munc13-4 protein) suffer from familial hemophagocytic lymphohistiocytosis type 3 (FHL3), a life-threatening immune and hyperinflammatory disorder. The only curative treatment is allogeneic hematopoietic stem cell (HSC) transplantation,...

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Главные авторы:	Tayebeh Soheili, Amandine Durand, Fernando E. Sepulveda, Julie Rivière, Chantal Lagresle-Peyrou, Hanem Sadek, Geneviève de Saint Basile, Samia Martin, Fulvio Mavilio, Marina Cavazzana, Isabelle André-Schmutz
Формат:	Artigo
Язык:	Inglês
Опубликовано:	Elsevier 2017-12-01
Серии:	Blood Advances
Online-ссылка:	http://www.sciencedirect.com/science/article/pii/S247395292030077X
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