Induced Pluripotent Stem Cell for the Study and Treatment of Sickle Cell Anemia

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Junqueira Reis, Luiza Cunha ; Picanco-Castro, Virginia ; Martins Fernandes Paes, Barbara Cristina ; Pereira, Olivia Ambrozini ; Gyuricza, Isabela Gerdes ; de Araujo, Fabiano Tofoli ; Morato-Marques, Mariana ; Moreira, Lilian Figueiredo ; Oliveira Costa, Everton de Brito ; Moreira dos Santos, Tÿlita Pollyanna ; Covas, Dimas Tadeu ; Pereira Carramaschi, Lygia da Veiga ; de Sousa Russo, Elisa Maria

• Document type: Journal article
• Source: STEM CELLS INTERNATIONAL; v. 2017, p. 30-pg., 2017-01-01.

Abstract

Sickle cell anemia (SCA) is a monogenic disease of high mortality, affecting millions of people worldwide. There is no broad, effective, and safe definitive treatment for SCA, so the palliative treatments are the most used. The establishment of an in vitro model allows better understanding of how the disease occurs, besides allowing the development of more effective tests and treatments. In this context, iPSC technology is a powerful tool for basic research and disease modeling, and a promise for finding and screening more effective and safe drugs, besides the possibility of use in regenerative medicine. This work obtained a model for study and treatment of SCA using iPSC. Then, episomal vectors were used for reprogramming peripheral blood mononuclear cells to obtain integration-free iPSC. Cells were collected from patients treated with hydroxyurea and without treatment. The iPSCP Bscd lines were characterized for pluripotent and differentiation potential. The iPSC lines were differentiated into HSC, so that we obtained a dynamic and efficient protocol of CD34+CD45+ cells production. We offer a valuable tool for a better understanding of how SCA occurs, in addition to making possible the development of more effective drugs and treatments and providing better understanding of widely used treatments, such as hydroxyurea. (AU)

• FAPESP's process: 13/08135-2 - CTC - Center for Cell-Based Therapy
• Grantee: Dimas Tadeu Covas
• Support Opportunities: Research Grants - Research, Innovation and Dissemination Centers - RIDC