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

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, although...

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Dades bibliogràfiques
Publicat a:Blood Adv
Autors principals: Soheili, Tayebeh, Durand, Amandine, Sepulveda, Fernando E., Rivière, Julie, Lagresle-Peyrou, Chantal, Sadek, Hanem, de Saint Basile, Geneviève, Martin, Samia, Mavilio, Fulvio, Cavazzana, Marina, André-Schmutz, Isabelle
Format: Artigo
Idioma:Inglês
Publicat: American Society of Hematology 2017
Matèries:
Gene Therapy
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5745141/
https://ncbi.nlm.nih.gov/pubmed/29296930
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2017012088
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