Gene therapy for primary immunodeficiencies: Part 2.
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.
San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET), Division of Regenerative Medicine, San Raffaele Scientific Institute, Milan, Italy. aiuti.alessandro@hsr.it
Alessandro
A
Aiuti
Rosa
R
Bacchetta
Reinhard
R
Seger
Anna
A
Villa
Marina
M
Cavazzana-Calvo
D000818:Animals; D002986:Clinical Trials as Topic / Q000639:trends; D017889:Exfoliation Syndrome / Q000235:genetics / Q000276:immunology / Q000628:therapy; D005544:Forecasting / Q000379:methods; D018014:Gene Transfer Techniques / Q000639:trends; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends*; D005822:Genetic Vectors / Q000235:genetics / Q000276:immunology / Q000627:therapeutic use; D006105:Granulomatous Disease, Chronic / Q000235:genetics / Q000276:immunology / Q000628:therapy; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000235:genetics* / Q000276:immunology / Q000628:therapy*; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000276:immunology / Q000628:therapy; D014923:Wiskott-Aldrich Syndrome / Q000235:genetics / Q000276:immunology / Q000628:therapy