Terapia CRISPR‑Cas9 na anemia falciforme e β‑talassemia

evidências clínicas e pré-clínicas em uma revisão sistemática

Authors

  • Ihury Jhonson Evangelista Alves de Lima
  • Elisa Mundim dos Santos Nunes Rosa
  • Genival José da Silva Neto
  • Gabriela Gonçalves Magalhães
  • Sofia Souza Bordignon
  • Nayane Peixoto Soares

Keywords:

CRISPR-Cas9, sickle cell disease, β-thalassemia, gene therapy

Abstract

Objective: This systematic review aimed to analyze clinical and preclinical evidence on the application of CRISPR-Cas9 gene therapy in sickle cell disease and β-thalassemia. The search was conducted in the PubMed, Embase, and Scielo databases, including articles published between 2015 and 2025. After screening using the PRISMA flowchart, 12 studies met the eligibility criteria. The results showed that CRISPR-Cas9 therapy, particularly with the use of CTX001, promoted sustained fetal hemoglobin production, elimination of vaso-occlusive crises, and transfusion independence. Preclinical studies reinforced the safety and efficacy of the technique, with low incidence of off-target effects. Despite these advances, challenges related to cost, access, and regulation still limit its widespread application. It is concluded that CRISPR-Cas9 represents a promising and potentially curative approach for hemoglobinopathies.

References

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Published

2026-03-24

How to Cite

Lima, I. J. E. A. de, Rosa, E. M. dos S. N., Silva Neto, G. J. da, Magalhães, G. G., Bordignon, S. S., & Soares, N. P. (2026). Terapia CRISPR‑Cas9 na anemia falciforme e β‑talassemia: evidências clínicas e pré-clínicas em uma revisão sistemática. Revista De Divulgação Científica Sena Aires, 15(Esp. 4), 61–67. Retrieved from https://rdcsa.emnuvens.com.br/revista/article/view/1144

Issue

Vol. 15 No. Esp. 4 (2026): REVISA

Section

Literature Review

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Copyright (c) 2026 Revista de Divulgação Científica Sena Aires

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