scholarly journals Gene Therapy for Primary Immunodeficiencies

10.5772/55594 ◽  
2013 ◽  
Author(s):  
Francisco Martin ◽  
Alejandra Gutierrez-Guerrero ◽  
Karim Benabdellah
Keyword(s):  
Gene Therapy ◽  
Primary Immunodeficiencies

Gene Therapy in Primary Immunodeficiencies

2013 ◽  
Vol 02 (01) ◽  
Author(s):  
Inês Rosinha ◽  
Laura Costa ◽  
Mara Pinto ◽  
Natália Barbosa ◽  
Patrícia Rosinha
Keyword(s):  
Gene Therapy ◽  
Primary Immunodeficiencies

Gene therapy for primary immunodeficiencies: Part 2

2012 ◽  
Vol 24 (5) ◽  
pp. 585-591 ◽  
Author(s):  
Alessandro Aiuti ◽  
Rosa Bacchetta ◽  
Reinhard Seger ◽  
Anna Villa ◽  
Marina Cavazzana-Calvo
Keyword(s):  
Gene Therapy ◽  
Primary Immunodeficiencies

scholarly journals Clinical Applications of Gene Therapy for Primary Immunodeficiencies

2015 ◽  
Vol 26 (4) ◽  
pp. 210-219 ◽  
Author(s):  
Maria Pia Cicalese ◽  
Alessandro Aiuti
Keyword(s):  
Gene Therapy ◽  
Clinical Applications ◽  
Primary Immunodeficiencies

Gene Therapy for Primary Immunodeficiencies

2010 ◽  
Vol 30 (2) ◽  
pp. 237-248 ◽  
Author(s):  
Alain Fischer ◽  
S. Hacein-Bey-Abina ◽  
M. Cavazanna-Calvo
Keyword(s):  
Gene Therapy ◽  
Primary Immunodeficiencies

scholarly journals Gene therapy with hematopoietic stem and progenitor cell for monogenic disorders: a systematic review and meta-analysis

2021 ◽  
Author(s):  
Francesca Tucci ◽  
Stefania Galimberti ◽  
Luigi Naldini ◽  
Maria G Valsecchi ◽  
Alessandro Aiuti
Keyword(s):  
Gene Therapy ◽  
Metabolic Diseases ◽  
Ex Vivo ◽  
Meta Analysis ◽  
Primary Immunodeficiencies ◽  
Hematopoietic Stem ◽  
Monogenic Disorders ◽  
Monogenic Diseases ◽  
Stem And Progenitor Cells ◽  
Ex Vivo Gene Therapy

Abstract To provide an assessment of the safety of ex-vivo gene therapy (GT) with hematopoietic stem and progenitor cells (HSPC), we reviewed in a systematic manner the literature on monogenic diseases to describe survival, genotoxicity and engraftment of gene corrected HSPC, across vector platforms and diseases. From 1995 to 2020, 55 trials for 14 diseases met inclusion criteria and 406 patients with primary immunodeficiencies (55.2%), metabolic diseases (17.0%), haemoglobinopathies (24.4%) and bone marrow failures (3.4%) were treated with gammaretroviral vector (γRV) (29.1%), self-inactivating γRV (2.2%) or lentiviral vectors (LV) (68.7%). The pooled overall incidence rate of death was 0.9 per 100 person-years of observation (PYO) (95%CI = 0.37–2.17). There were 21 genotoxic events out of 1504.02 PYO. All these events occurred in γRV trials (0.99 events per 100 PYO, 95%CI = 0.18–5.43) for primary immunodeficiencies. Pooled rate of engraftment was 86.1% (95%CI = 66.9–95.0%) for γRV and 99.0% (95%CI = 95.1–99.8%) for LV HSPC-GT (p = 0.002). A comprehensive meta-analysis on HSPC-GT showed stable reconstitution of haematopoiesis in most recipients with superior engraftment and safer profile in patients receiving LV-transduced HSPC.

Gene therapy for primary immunodeficiencies: part 1

2012 ◽  
Vol 24 (5) ◽  
pp. 580-584 ◽  
Author(s):  
Marina Cavazzana-Calvo ◽  
Alain Fischer ◽  
Salima Hacein-Bey-Abina ◽  
Alessandro Aiuti
Keyword(s):  
Gene Therapy ◽  
Primary Immunodeficiencies

Hematopoietic stem cell gene therapy for the cure of blood diseases: primary immunodeficiencies

2018 ◽  
Vol 29 (4) ◽  
pp. 755-764 ◽  
Author(s):  
Cristina Cifaldi ◽  
Francesca Ferrua ◽  
Alessandro Aiuti ◽  
Caterina Cancrini
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Primary Immunodeficiencies ◽  
Hematopoietic Stem ◽  
Blood Diseases ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

Gene therapy of primary immunodeficiencies

1998 ◽  
Vol 19 (4) ◽  
pp. 493-508 ◽  
Author(s):  
Fabio Candotti ◽  
R. Michael Blaese
Keyword(s):  
Gene Therapy ◽  
Primary Immunodeficiencies
Sign in / Sign up

Export Citation Format

Share Document