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GENERATION OF INTEGRATION-FREE IPS CELL LINES FROM THREE SICKLE CELL DISEASE PATIENTS FROM THE STATE OF BAHIA, BRAZIL
Autor
Martins, Gabriele Louise Soares
Paredes, Bruno Diaz
Azevedo, Carine Machado
Sampaio, Gabriela Louise de Almeida
Nonaka, Carolina Kymie Vasques
Cavalcante, Bruno Raphael Ribeiro
Silva, Katia Nunes da
Pereira, Ciro Silveira e
Soares, Milena Botelho Pereira
Santos, Ricardo Ribeiro dos
Souza, Bruno Solano de Freitas
Paredes, Bruno Diaz
Azevedo, Carine Machado
Sampaio, Gabriela Louise de Almeida
Nonaka, Carolina Kymie Vasques
Cavalcante, Bruno Raphael Ribeiro
Silva, Katia Nunes da
Pereira, Ciro Silveira e
Soares, Milena Botelho Pereira
Santos, Ricardo Ribeiro dos
Souza, Bruno Solano de Freitas
Afiliación
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil / Fundação Oswaldo Cruz. Instituto Gonçalo Moniz. Salvador, BA, Brasil.
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil / National Institute of Science and Technology for Regenerative Medicine. Rio de Janeiro, RJ, Brazil.
Fundação Oswaldo Cruz. Instituto Gonçalo Moniz. Salvador, BA, Brasil.
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil / Fundação Oswaldo Cruz. Instituto Gonçalo Moniz. Salvador, BA, Brasil.
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil / Fundação Oswaldo Cruz. Instituto Gonçalo Moniz. Salvador, BA, Brasil.
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil / National Institute of Science and Technology for Regenerative Medicine. Rio de Janeiro, RJ, Brazil.
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil / Fundação Oswaldo Cruz. Instituto Gonçalo Moniz. Salvador, BA, Brasil / National Institute of Science and Technology for Regenerative Medicine. Rio de Janeiro, RJ, Brazil.
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil / Fundação Oswaldo Cruz. Instituto Gonçalo Moniz. Salvador, BA, Brasil / National Institute of Science and Technology for Regenerative Medicine. Rio de Janeiro, RJ, Brazil.
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil / Fundação Oswaldo Cruz. Instituto Gonçalo Moniz. Salvador, BA, Brasil / National Institute of Science and Technology for Regenerative Medicine. Rio de Janeiro, RJ, Brazil.
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil / National Institute of Science and Technology for Regenerative Medicine. Rio de Janeiro, RJ, Brazil.
Fundação Oswaldo Cruz. Instituto Gonçalo Moniz. Salvador, BA, Brasil.
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil / Fundação Oswaldo Cruz. Instituto Gonçalo Moniz. Salvador, BA, Brasil.
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil / Fundação Oswaldo Cruz. Instituto Gonçalo Moniz. Salvador, BA, Brasil.
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil / National Institute of Science and Technology for Regenerative Medicine. Rio de Janeiro, RJ, Brazil.
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil / Fundação Oswaldo Cruz. Instituto Gonçalo Moniz. Salvador, BA, Brasil / National Institute of Science and Technology for Regenerative Medicine. Rio de Janeiro, RJ, Brazil.
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil / Fundação Oswaldo Cruz. Instituto Gonçalo Moniz. Salvador, BA, Brasil / National Institute of Science and Technology for Regenerative Medicine. Rio de Janeiro, RJ, Brazil.
São Rafael Hospital. Center for Biotechnology and Cell Therapy. Salvador, BA, Brazil / Fundação Oswaldo Cruz. Instituto Gonçalo Moniz. Salvador, BA, Brasil / National Institute of Science and Technology for Regenerative Medicine. Rio de Janeiro, RJ, Brazil.
Resumen en ingles
Sickle cell disease (SCD) is one of the most prevalent and severe monogenetic disorders, affecting several million people around the world. Clinical manifestations
and complications of the disease include sickle cell pain crisis, silent cerebral infarct, stroke, nephropathy and early death. In this study, we generated induced
pluripotent stem cell (iPSC) lines from three homozygous SCD patients from the state of Bahia, Brazil, where SCD is highly prevalent. Peripheral blood mononuclear
cells were collected and erythroblasts were expanded for cell reprogramming with the use of non-integrative episomal vectors. The generated iPSC lines expressed
high levels of pluripotency markers, presented a normal karyotype and were able to differentiate into the three germ layers in embryoid body spontaneous differentiation
assays. Moreover, the expression of the episomal vectors was lost in all iPSC lines after 15 passages. These iPSC lines may help increasing the knowledge
about SCD pathogenesis and can be a useful tool for drug testing and gene editing studies.
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