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Lentiviral vectors for the treatment of primary immunodeficiencies.
J Inherit Metab Dis. 2014 Jul; 37(4):525-33.JI

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.

Authors+Show Affiliations

Department of Pediatrics, Children's Hospital Bambino Gesù and University of Rome Tor Vergata School of Medicine, Rome, Italy.No affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't
Review

Language

eng

PubMed ID

24619149

Citation

Farinelli, Giada, et al. "Lentiviral Vectors for the Treatment of Primary Immunodeficiencies." Journal of Inherited Metabolic Disease, vol. 37, no. 4, 2014, pp. 525-33.
Farinelli G, Capo V, Scaramuzza S, et al. Lentiviral vectors for the treatment of primary immunodeficiencies. J Inherit Metab Dis. 2014;37(4):525-33.
Farinelli, G., Capo, V., Scaramuzza, S., & Aiuti, A. (2014). Lentiviral vectors for the treatment of primary immunodeficiencies. Journal of Inherited Metabolic Disease, 37(4), 525-33. https://doi.org/10.1007/s10545-014-9690-y
Farinelli G, et al. Lentiviral Vectors for the Treatment of Primary Immunodeficiencies. J Inherit Metab Dis. 2014;37(4):525-33. PubMed PMID: 24619149.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Lentiviral vectors for the treatment of primary immunodeficiencies. AU - Farinelli,Giada, AU - Capo,Valentina, AU - Scaramuzza,Samantha, AU - Aiuti,Alessandro, Y1 - 2014/03/12/ PY - 2013/12/27/received PY - 2014/02/07/accepted PY - 2014/02/06/revised PY - 2014/3/13/entrez PY - 2014/3/13/pubmed PY - 2015/5/13/medline SP - 525 EP - 33 JF - Journal of inherited metabolic disease JO - J. Inherit. Metab. Dis. VL - 37 IS - 4 N2 - 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. SN - 1573-2665 UR - https://www.unboundmedicine.com/medline/citation/24619149/Lentiviral_vectors_for_the_treatment_of_primary_immunodeficiencies_ L2 - https://doi.org/10.1007/s10545-014-9690-y DB - PRIME DP - Unbound Medicine ER -