T-cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents hemophagocytic lymphohistiocytosis manifestations

BACKGROUND: Mutations in the perforin 1 (PRF1) gene account for up to 58% of familial hemophagocytic lymphohistiocytosis syndromes. The resulting defects in effector cell cytotoxicity lead to hypercytokinemia and hyperactivation with inflammation in various organs. OBJECTIVE: We sought to determine...

Celý popis

Uloženo v:
Podrobná bibliografie
Vydáno v:J Allergy Clin Immunol
Hlavní autoři: Ghosh, Sujal, Carmo, Marlene, Calero-Garcia, Miguel, Ricciardelli, Ida, Bustamante Ogando, Juan Carlos, Blundell, Michael P., Schambach, Axel, Ashton-Rickardt, Philip G., Booth, Claire, Ehl, Stephan, Lehmberg, Kai, Thrasher, Adrian J., Gaspar, H. Bobby
Médium: Artigo
Jazyk:Inglês
Vydáno: Mosby 2018
Témata:
Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6127027/
https://ncbi.nlm.nih.gov/pubmed/29355678
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jaci.2017.11.050
Tagy: Přidat tag
Žádné tagy, Buďte první, kdo otaguje tento záznam!

Podobné jednotky