Article

Immunologic Research

, Volume 43, Issue 1, pp 223-242

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

  • Suk See De RavinAffiliated withGenetic Immunotherapy, Laboratory of Host Defense, National Institutes of Health Email author 
  • , Harry L. MalechAffiliated withGenetic Immunotherapy, Laboratory of Host Defense, National Institutes of Health

Rent the article at a discount

Rent now

* Final gross prices may vary according to local VAT.

Get Access

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 immunodeficiency Hematopoietic stem cell transplant Gene therapy Haploidentical T cell recombination excision circles T cell receptor Vbeta spectratype