Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation


Capo V., Castiello M. C. , Fontana E., Penna S., Bosticardo M., Draghici E., ...More

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, vol.142, no.3, pp.928-949, 2018 (Journal Indexed in SCI) identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 142 Issue: 3
  • Publication Date: 2018
  • Doi Number: 10.1016/j.jaci.2017.11.015
  • Title of Journal : JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
  • Page Numbers: pp.928-949

Abstract

Background: Omenn syndrome (OS) is a rare severe combined immunodeficiency associated with autoimmunity and caused by defects in lymphoid-specific V(D)J recombination. Most patients carry hypomorphic mutations in recombination-activating gene (RAG) 1 or 2. Hematopoietic stem cell transplantation is the standard treatment; however, gene therapy (GT) might represent a valid alternative, especially for patients lacking a matched donor.