TY - JOUR
T1 - Gene therapy for primary immunodeficiencies
T2 - Part 2
AU - Aiuti, Alessandro
AU - Bacchetta, Rosa
AU - Seger, Reinhard
AU - Villa, Anna
AU - Cavazzana-Calvo, Marina
PY - 2012/10
Y1 - 2012/10
N2 - Gene therapy has become an attractive alternative therapeutic strategy to allogeneic transplant for primary immunodeficiencies (PIDs) owing to known genetic defects. Clinical trials using gammaretroviral vectors have demonstrated the proof of principle of gene therapy for Wiskott-Aldrich syndrome (WAS) and chronic granulomatous disease (CGD), but have also highlighted limitations of the technology. New strategies based on vectors that can achieve more robust correction with less risk of insertional mutagenesis are being developed. In this review we present the status of gene therapy for WAS and CGD, and discuss the emerging application of similar strategies to a broader range of PIDs, such as IPEX syndrome.
AB - Gene therapy has become an attractive alternative therapeutic strategy to allogeneic transplant for primary immunodeficiencies (PIDs) owing to known genetic defects. Clinical trials using gammaretroviral vectors have demonstrated the proof of principle of gene therapy for Wiskott-Aldrich syndrome (WAS) and chronic granulomatous disease (CGD), but have also highlighted limitations of the technology. New strategies based on vectors that can achieve more robust correction with less risk of insertional mutagenesis are being developed. In this review we present the status of gene therapy for WAS and CGD, and discuss the emerging application of similar strategies to a broader range of PIDs, such as IPEX syndrome.
UR - http://www.scopus.com/inward/record.url?scp=84867746098&partnerID=8YFLogxK
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U2 - 10.1016/j.coi.2012.07.012
DO - 10.1016/j.coi.2012.07.012
M3 - Article
C2 - 22909900
AN - SCOPUS:84867746098
VL - 24
SP - 585
EP - 591
JO - Current Opinion in Immunology
JF - Current Opinion in Immunology
SN - 0952-7915
IS - 5
ER -