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Development of a pCCLchim lentiviral vector for gene therapy of patients with chronic granulomatous disease due to p47-phox deficiency

Grant number: 15/13110-4
Support type:Scholarships abroad - Research Internship - Post-doctor
Effective date (Start): January 04, 2016
Effective date (End): December 14, 2016
Field of knowledge:Biological Sciences - Genetics - Human and Medical Genetics
Principal researcher:Antonio Condino Neto
Grantee:Walmir Cutrim Aragão Filho
Supervisor abroad: Adrian Thrasher
Home Institution: Instituto de Ciências Biomédicas (ICB). Universidade de São Paulo (USP). São Paulo , SP, Brazil
Research place: University College London (UCL), England  
Associated to the scholarship:14/01962-3 - Development of a pCCLchim lentiviral vector for gene therapy of patients with chronic granulomatous disease due to p47-phox deficiency, BP.PD


The NADPH oxidase system is an enzymatic complex that generates superoxide. It is formed by membrane components, gp91-phox and p22-phox subunits (flavocytochrome b558), and cytoplasmic components (p47-phox, p67-phox and p40-phox). Defects in the expression of these components lead to Chronic Granulomatous Disease (CGD), a severe primary immunodeficiency characterized by the early onset of severe and recurrent infections. To date, allogeneic transplantation of hematopoietic stem cells has been the treatment of choice for patients affected by this disease, however, it is only available to a third of the patients due to HLA (human leukocyte antigen) compatibility issues. Gene therapy can fill this treatment gap once primary patient cells can be genetically corrected and transplanted back to the same donor, with the possibility of cure. Gene therapy protocols for CGD based on gammaretroviral vectors have not shown good results. In this context, pre-clinical trials have shown efficiency in correcting the expression of NADPH oxidase components by the substitution of gammaretroviral vectors by lentiviral vectors. Considering the importance of developing an effective gene therapy for patients with CGD and the promising results found in the literature through the use of lentiviral vectors, in the present BEPE project we will test the efficiency of a new lentiviral vector for correction of the expression of the NADPH oxidase p47-phox component based on the chimeric vector pCCLchim, which is well characterized by Santilli and colleagues (2011).

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Scientific publications
(References retrieved automatically from Web of Science and SciELO through information on FAPESP grants and their corresponding numbers as mentioned in the publications by the authors)
SCHEJTMAN, ANDREA; ARAGAO-FILHO, WALMIR CUTRIM; CLARE, SIMON; ZINICOLA, MARTA; WEISSER, MAREN; BURNS, SIOBHAN O.; BOOTH, CLAIRE; GASPAR, HUBERT B.; THOMAS, DAVID C.; CONDINO-NETO, ANTONIO; et al. Lentiviral gene therapy rescues p47(phox)chronic granulomatous disease and the ability to fightSalmonellainfection in mice. Gene Therapy, v. 27, n. 9, . (15/13110-4, 14/01962-3)

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