Immunologic Research

, Volume 43, Issue 1, pp 223–242

Partially corrected X-linked severe combined immunodeficiency: long-term problems and treatment options

Article

DOI: 10.1007/s12026-008-8073-6

Cite this article as:
De Ravin, S.S. & Malech, H.L. Immunol Res (2009) 43: 223. doi:10.1007/s12026-008-8073-6

Abstract

Rapid progress has been made from the identification of the molecular defects causing X-linked severe combined immune deficiency (X-SCID) to the development of cutting-edge therapeutic approaches such as hematopoietic stem cell transplant and gene therapy for XSCID. Successful treatment of XSCID has created a new population of patients, many of whom are now adolescents and young adults and are facing a variety of chronic problems secondary to partial correction of their underlying disease. This review focuses on the clinical challenges facing these patients (and their caregivers) and provides an overview of some of the treatment options available, including gene therapy.

Keywords

Severe combined immunodeficiencyHematopoietic stem cell transplantGene therapyHaploidenticalT cell recombination excision circlesT cell receptor Vbeta spectratype

Copyright information

© Humana Press Inc. 2008

Authors and Affiliations

  1. 1.Genetic Immunotherapy, Laboratory of Host DefenseNational Institutes of HealthBethesda USA