Academic Journals Database
Disseminating quality controlled scientific knowledge

Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency

ADD TO MY LIST
 
Author(s): Claudia A Montiel-Equihua | Adrian J Thrasher | H Bobby Gaspar

Journal: Stem Cells and Cloning : Advances and Applications
ISSN 1178-6957

Volume: 2010;
Issue: default;
Start page: 1;
Date: 2009;
Original page

ABSTRACT
Claudia A Montiel-Equihua, Adrian J Thrasher, H Bobby GasparCentre for Immunodeficiency, Molecular Immunology Unit, UCL Institute of Child Health, London, UKAbstract: The history of stem cell gene therapy is strongly linked to the development of gene therapy for severe combined immunodeficiencies (SCID) and especially adenosine deaminase (ADA)-deficient SCID. Here we discuss the developments achieved in over two decades of clinical and laboratory research that led to the establishment of a protocol for the autologous transplant of retroviral vector-mediated gene-modified hematopoietic stem cells, which has proved to be both successful and, to date, safe. Patients in trials in three different countries have shown long-term immunological and metabolic correction. Nevertheless, improvements to the safety profile of viral vectors are underway and will undoubtedly reinforce the position of stem cell gene therapy as a treatment option for ADA-SCID.Keywords: adenosine deaminase, severe combined immunodeficiency, gene therapy, hematopoietic stem cell, retrovirus, clinical trial
Save time & money - Smart Internet Solutions      Why do you need a reservation system?