Gene therapy for primary immunodeficiencies: part 1

Highlights ► Gene therapy provided a clinical benefit to most of the 60 patients affected by ADA-deficiency or SCID-X1. ► Gene therapy can be a valuable alternative to allogeneic hematopoietic stem cell transplantation. ► Insertional oncogenesis still represents the major issue to solve. ► Homologou...

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Veröffentlicht in:	Current opinion in immunology 2012-10, Vol.24 (5), p.580-584
Hauptverfasser:	Cavazzana-Calvo, Marina, Fischer, Alain, Hacein-Bey-Abina, Salima, Aiuti, Alessandro
Format:	Artikel
Sprache:	eng
Schlagworte:	Adenosine Deaminase - deficiency Adenosine Deaminase - genetics Allergy and Immunology Animals Clinical Trials as Topic - methods Clinical Trials as Topic - trends Disease Models, Animal Genetic Therapy - methods Genetic Therapy - trends Hematopoietic Stem Cell Transplantation - methods Hematopoietic Stem Cell Transplantation - trends Humans Immunologic Deficiency Syndromes - genetics Immunologic Deficiency Syndromes - immunology Immunologic Deficiency Syndromes - therapy X-Linked Combined Immunodeficiency Diseases - genetics X-Linked Combined Immunodeficiency Diseases - immunology X-Linked Combined Immunodeficiency Diseases - therapy
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Beschreibung
Zusammenfassung:	Highlights ► Gene therapy provided a clinical benefit to most of the 60 patients affected by ADA-deficiency or SCID-X1. ► Gene therapy can be a valuable alternative to allogeneic hematopoietic stem cell transplantation. ► Insertional oncogenesis still represents the major issue to solve. ► Homologous recombination is rapidly improving to edit gene mutations.
ISSN:	0952-7915 1879-0372
DOI:	10.1016/j.coi.2012.08.008