RNA DE INTERFERÊNCIA COMO ESTRATÉGIA TERAPÊUTICA
DOI:
https://doi.org/10.36557/2674-8169.2026v8n2p5-18Palavras-chave:
RNA interferente, Silenciamento gênico, Nanopartículas lipídicas, Biofarmacologia translocacionalResumo
Introdução: A descoberta da RNAi revelou um mecanismo natural de silenciamento gênico onde moléculas de RNA degradam o RNA mensageiro alvo. Isso quebrou o dogma do RNA como mero intermediário e abriu uma nova fronteira terapêutica. Objetivos: Analisar os mecanismos moleculares da RNAi, a evolução das tecnologias de entrega e o atual estágio de suas aplicações clínicas. Metodologia: Revisão narrativa focada na via canônica (Dicer/RISC), na comparação entre vetores virais e não virais (LNPs e GalNAc) e no histórico de aprovações regulatórias. Conclusão: A tecnologia é um sucesso clínico validado por fármacos como o Patisiran, permitindo tratar doenças antes incuráveis. O desafio atual é expandir a entrega para além do fígado e integrar a técnica a novas ferramentas como o CRISPR.
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Copyright (c) 2026 Fernando Malachias de Andrade Bergamo, Gustavo Bueno Kincheski, Rafael Shenji Kitamura, Débora Marioto
Este trabalho está licenciado sob uma licença Creative Commons Attribution 4.0 International License.
Os autores são detentores dos direitos autorais mediante uma licença CCBY 4.0.
