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Article

Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Apr 2015

Carmo Marlene, Risma Kimberly A, Arumugam Paritha, Tiwari Swati, Hontz Adrianne E, Montiel-Equihua Claudia A, Alonso-Ferrero Maria E, Blundell Michael P, Schambach Axel, Baum Christopher, Malik Punam, Thrasher Adrian J, Jordan Michael B, Gaspar H Bobby

Abstract excerpt

Defects in perforin lead to the failure of T and NK cell cytotoxicity, hypercytokinemia, and the immune dysregulatory condition known as familial hemophagocytic lymphohistiocytosis (FHL). The only curative treatment is allogeneic hematopoietic stem cell transplantation which carries substantial risks. We used lentiviral vectors (LV) expressing the human perforin gene, under the transcriptional control of the...

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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

Share to Gene Transfer Techniques

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Share to Killer Cells, Natural

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Source

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Identifiers

PMID: 25523759
DOI: 10.1038/mt.2014.242
PMCID: PMC4395774

Provenance

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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