Journal of Primary & Acquired Immunodeficiency ResearchISSN: 2324-853X

Review Article, J Immunodefic Disor Vol: 2 Issue: 1

Gene Therapy in Primary Immunodeficiencies

Inês Rosinha, Laura Costa*, Mara Pinto, Natália Barbosa and Patrícia Rosinha
Faculty of Medicine, University of Porto, Portugal
Corresponding author : Laura Costa
Faculty of Medicine, University of Porto, Portugal
E-mail: [email protected]
Received: February 28, 2013 Accepted: May 30, 2013 Published: June 07, 2013
Citation: Rosinha I, Costa L, Pinto M, Barbosa N, Rosinha P (2012) Gene Therapy in Primary Immunodeficiencies. J Immunodefic Disor 2:1. doi:10.4172/2324-853X.1000103

Abstract

Gene Therapy in Primary Immunodeficiencies

Primary immunodeficiencies (PIDs) result from inherited mutations in genes involved in the production, function or survival of specific elements of the immune system (T, B, NK lymphocytes, neutrophils, antigen-presenting cells). Knowing that these elements derive from pluripotent hematopoietic stem cells, it has been tried to use allogeneic hematopoietic stem cell transplantation (HSCT) as an alternative for patients with a PID, which has proven to be an effective  therapeutic approach leading to immune restoration.

Keywords: Primary immunodeficiencies; Gene therapy; Hematopoietic stem cell transplantation; ADA-SCID; SCID-X1; Chronic granulomatous disease; Wiskott-Aldrich syndrome; Insertional mutagenesis

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