Terapias baseadas em mRNA: estratégias em formulação, sistemas de entrega e desafios translacionais para a aplicação clínica
DOI:
https://doi.org/10.61411/rsc2026136519Palavras-chave:
Terapias com mRNA; nanopartículas lipídicas; sistemas de liberação de fármacos; formulação farmacêutica.Resumo
As terapias baseadas em RNA mensageiro (mRNA) emergiram como uma plataforma transformadora na biotecnologia farmacêutica, impulsionada por avanços em engenharia molecular e sistemas de entrega de fármacos. Esta revisão narrativa crítica teve como objetivo discutir as principais estratégias de formulação, sistemas de entrega e aplicações clínicas das terapias baseadas em mRNA, bem como analisar os desafios translacionais para sua consolidação na prática clínica. O estudo foi conduzido com busca em PubMed, Scopus, ScienceDirect e Web of Science, de janeiro de 2018 a dezembro de 2025, incluindo referências clássicas relevantes. Foram utilizados termos como “messenger RNA”, “mRNA therapeutics”, “lipid nanoparticles” e “RNA delivery systems”. Incluíram-se artigos originais, revisões, ensaios clínicos e documentos regulatórios; excluíram-se duplicatas, resumos sem texto completo e estudos fora do escopo. A análise crítica integrou dados experimentais, perspectivas de especialistas e tendências regulatórias. O design do mRNA - incluindo modificações nucleotídicas, otimização de códons e engenharia de regiões não traduzidas - melhora estabilidade, tradução e tolerabilidade imunológica. Sistemas de entrega, como nanopartículas lipídicas (LNPs), polímeros, vesículas extracelulares e nanossistemas híbridos, são cruciais para eficácia terapêutica. Além de vacinas contra doenças infecciosas, o mRNA é aplicado em oncologia, doenças genéticas raras e medicina personalizada, considerando formulação, escalabilidade e aspectos regulatórios. O mRNA é uma plataforma terapêutica versátil, cujo impacto futuro depende de inovação contínua em formulação, controle de qualidade e sistemas de entrega, consolidando seu papel na medicina moderna.
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