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Article

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

Human gene therapy - 1 Oct 2016

Tiwari Swati, Hontz Adrianne, Terrell Catherine E, Arumugam Paritha, Carmo Marlene, Risma Kimberly, Jordan Michael, Malik Punam

Abstract excerpt

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 mortality, making gene therapy an attractive alternative approach for HLH. We reported that perforin expression...

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Identifiers

PMID: 27471778
DOI: 10.1089/hum.2016.065
PMCID: PMC5749578

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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