Lentiviral Gene Transfer Corrects Immune Abnormalities in XIAP Deficiency.

ABSTRACT:

Background

X-linked inhibitor of apoptosis protein (XIAP) deficiency is a severe immunodeficiency with clinical features including hemophagocytic lymphohistiocytosis (HLH) and inflammatory bowel disease (IBD) due to defective NOD2 responses. Management includes immunomodulatory therapies and hematopoietic stem cell transplant (HSCT). However, this cohort is particularly susceptible to the chemotherapeutic regimens and acutely affected by graft-vs-host disease (GvHD), driving poor long-term survival in transplanted patients. Autologous HSC gene therapy could offer an alternative treatment option and would abrogate the risks of alloreactivity.

Methods

Hematopoietic progenitor (Lin^-ve) cells from XIAP^y/- mice were transduced with a lentiviral vector encodi

SUBMITTER: Topal J 

PROVIDER: S-EPMC9892131 | biostudies-literature | 2023 Feb

REPOSITORIES: biostudies-literature