Journal of Inherited Metabolic Disease

, Volume 37, Issue 4, pp 525–533

Lentiviral vectors for the treatment of primary immunodeficiencies

  • Giada Farinelli
  • Valentina Capo
  • Samantha Scaramuzza
  • Alessandro Aiuti
ICIEM Symposium 2013

DOI: 10.1007/s10545-014-9690-y

Cite this article as:
Farinelli, G., Capo, V., Scaramuzza, S. et al. J Inherit Metab Dis (2014) 37: 525. doi:10.1007/s10545-014-9690-y

Abstract

In the last years important progress has been made in the treatment of several primary immunodeficiency disorders (PIDs) with gene therapy. Hematopoietic stem cell (HSC) gene therapy indeed represents a valid alternative to conventional transplantation when a compatible donor is not available and recent success confirmed the great potential of this approach. First clinical trials performed with gamma retroviral vectors were promising and guaranteed clinical benefits to the patients. On the other hand, the outcome of severe adverse events as the development of hematological abnormalities highlighted the necessity to develop a safer platform to deliver the therapeutic gene. Self-inactivating (SIN) lentiviral vectors (LVVs) were studied to overcome this hurdle through their preferable integration pattern into the host genome. In this review, we describe the recent advancements achieved both in vitro and at preclinical level with LVVs for the treatment of Wiskott-Aldrich syndrome (WAS), chronic granulomatous disease (CGD), ADA deficiency (ADA-SCID), Artemis deficiency, RAG1/2 deficiency, X-linked severe combined immunodeficiency (γchain deficiency, SCIDX1), X-linked lymphoproliferative disease (XLP) and immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome.

Copyright information

© SSIEM and Springer Science+Business Media Dordrecht 2014

Authors and Affiliations

  • Giada Farinelli
    • 1
  • Valentina Capo
    • 1
  • Samantha Scaramuzza
    • 2
  • Alessandro Aiuti
    • 1
    • 2
    • 3
    • 4
  1. 1.Department of PediatricsChildren’s Hospital Bambino Gesù and University of Rome Tor Vergata School of MedicineRomeItaly
  2. 2.San Raffaele Telethon Institute for Gene Therapy (TIGET)Scientific Institute HS RaffaeleMilanItaly
  3. 3.Dip. di Medicina dei SistemiUniversity of Rome Tor VergataRomaItaly
  4. 4.HSR-TIGET, Scientific Institute San RaffaeleMilanoItaly