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National production of genetically modified pigs for organ xenotransplantation in humans

Grant number: 18/14275-5
Support type:Research Grants - Research Partnership for Technological Innovation - PITE
Duration: October 01, 2019 - September 30, 2022
Field of knowledge:Biological Sciences - Genetics
Principal Investigator:Silvano Mario Attilio Raia
Grantee:Silvano Mario Attilio Raia
Home Institution: Faculdade de Medicina (FM). Universidade de São Paulo (USP). São Paulo , SP, Brazil
Company: EMS S A
CNAE: Atividades de atenção à saúde humana não especificadas anteriormente
CNAE: Atividades de atenção à saúde humana não especificadas anteriormente
City: São PauloHortolândia
Co-Principal Investigators:Jorge Elias Kalil Filho ; Maria Rita dos Santos e Passos Bueno ; Mayana Zatz
Assoc. researchers:Ernesto da Silveira Goulart Guimarães ; Luciano Abreu Brito ; Luiz Carlos de Caires Júnior
Associated grant(s):19/09467-5 - Multi-User Equipment approved in grant 18/14275-5: Micromanipulator - equipment for nuclear transfer, AP.EMU
19/09474-1 - Multi-User Equipment approved in grant 18/14275-5: Nucleofector 4D - equipment used for genomic editing, AP.EMU

Abstract

Motivated by the growing need for transplantation-available organs and the latest developments in genomic editing technologies such as the CRISPR-Cas9 system, significant progress in the efficiency and safety of xenotransplantation techniques has been achieved in the last two years. Genetically engineered pigs developed to prevent receptor hyperacute immune rejection have been successfully developed in other countries. However, the possibility of cross-infection of pathogens such as PERVs (porcine endogenous retrovirus) must be controlled before the starting of therapeutic trials in humans. This project proposes the innovative national production of genetically modified pigs where genes known as inducers of hyperacute rejection (GGTA1 / CMAH / ²4GalNT2) will be knocked out. Furthermore, the copies of the PERV pol gene will be inactivated from the porcine genomic DNA. To this end, this project associates geneticists, immunologists, surgeons and clinicians with known experience and credibility in their respective area of knowledge for the development of a national biotechnological based product. At the very end, we aim to provide the population in waiting list for transplants a viable alternative and definitive therapy, thus shortening the suffering of the patients and their families. Furthermore, besides technological aspects, the viability of xenotransplantation in Brazil will require great efforts to conciliate ethical, religious and legal aspects. On this respect, our previous experience as a pioneer of intervivos liver transplantation in the world in the 80s will be extremely valuable. (AU)

Articles published in Agência FAPESP Newsletter about the research grant
Brazil will have the technology to transplant pig organs into humans  
Articles published in other media outlets (5 total):
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Santa Casa e USP buscam formas de usar rins de porcos em transplantes 
Santa Casa e USP buscam formas de usar rins de porcos em transplantes 
Brasileños desarrollan una técnica para descontaminar órganos trasplantados de virus del donante 
Técnica brasileira visa ao transplante de órgãos de porcos para humanos 
Técnica desenvolvida no Brasil visa ao transplante de órgãos de porcos para humanos