Over 60 patients affected by SCID due to IL2RG deficiency (SCID-X1) or adenosine deaminase (ADA)-SCID have received hematopoietic stem cell gene therapy in the past 15 years using gammaretroviral vectors, resulting in immune reconstitution and clinical benefit in the majority of them. However, the occurrence of insertional oncogenesis in the SCID-X1 trials has led to the development of new clinical trials based on integrating vectors with improved safety design as well as investigation on new technologies for highly efficient gene targeting and site-specific gene editing. Here we will present the experience and perspectives of gene therapy for SCID-X1 and ADA-SCID and discuss the pros and cons of gene therapy in comparison to allogeneic transplantation.

Cavazzana Calvo, M., Fischer, A., Hacein Bey Abina, S., Aiuti, A. (2012). Gene therapy for primary immunodeficiencies: Part 1. CURRENT OPINION IN IMMUNOLOGY, 24(5), 580-584 [10.1016/j.coi.2012.08.008].

Gene therapy for primary immunodeficiencies: Part 1

AIUTI, ALESSANDRO
2012-10-01

Abstract

Over 60 patients affected by SCID due to IL2RG deficiency (SCID-X1) or adenosine deaminase (ADA)-SCID have received hematopoietic stem cell gene therapy in the past 15 years using gammaretroviral vectors, resulting in immune reconstitution and clinical benefit in the majority of them. However, the occurrence of insertional oncogenesis in the SCID-X1 trials has led to the development of new clinical trials based on integrating vectors with improved safety design as well as investigation on new technologies for highly efficient gene targeting and site-specific gene editing. Here we will present the experience and perspectives of gene therapy for SCID-X1 and ADA-SCID and discuss the pros and cons of gene therapy in comparison to allogeneic transplantation.
ott-2012
Pubblicato
Rilevanza internazionale
Articolo
Esperti anonimi
Settore MED/38 - PEDIATRIA GENERALE E SPECIALISTICA
English
Animals; Humans; Hematopoietic Stem Cell Transplantation; Clinical Trials as Topic; X-Linked Combined Immunodeficiency Diseases; Disease Models, Animal; Genetic Therapy; Adenosine Deaminase; Immunologic Deficiency Syndromes
Cavazzana Calvo, M., Fischer, A., Hacein Bey Abina, S., Aiuti, A. (2012). Gene therapy for primary immunodeficiencies: Part 1. CURRENT OPINION IN IMMUNOLOGY, 24(5), 580-584 [10.1016/j.coi.2012.08.008].
Cavazzana Calvo, M; Fischer, A; Hacein Bey Abina, S; Aiuti, A
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2108/78451
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