High Level of Perforin Expression Is Required for Effective Correction of Hemophagocytic Lymphohistiocytosis

Perforin-1 mutations result in a potentially fatal hemophagocytic lymphohistiocytosis (HLH) with heightened immune activation, hypercytokinemia, pancytopenia, and end-organ damage. At present, hematopoietic stem cell (HSC) transplantation is curative, but limited by donor availability and associated...

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Bibliographische Detailangaben
Veröffentlicht in:Hum Gene Ther
Hauptverfasser: Tiwari, Swati, Hontz, Adrianne, Terrell, Catherine E., Arumugam, Paritha, Carmo, Marlene, Risma, Kimberly, Jordan, Michael, Malik, Punam
Format: Artigo
Sprache:Inglês
Veröffentlicht: Mary Ann Liebert, Inc. 2016
Schlagworte:
Research Articles
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5749578/
https://ncbi.nlm.nih.gov/pubmed/27471778
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2016.065
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