A nova grande promessa da inovação em fármacos: RNA interferência saindo do laboratório para a clínica

Abstract

A descoberta de que nossas células dispõem de um mecanismo de silenciamento gênico empregando RNA interferência ainda é muito recente. Apesar disso, em menos de uma década a investigação científica já alcançou progresso, suficiente para muito brevemente nos apropriarmos desse conhecimento com fins terapêuticos. Duplexes de RNA são potenciais fármacos e há investimentos altos nessa nova abordagem. Aparentemente, a promessa de terapia gênica parece finalmente atingir sua maturidade com essas novas ferramentas.<br>The discovery of gene silencing mechanisms in our own cells using RNA interference is very recent. However, in less than a decade, the scientific investigation have progressed enough to make us see that, very soon, we will use this knowledge for therapeutic purposes. RNA duplexes are potential pharmaceutical drugs and there are high investments in this new strategy. The promising gene therapy seems to finally reach maturity with these new tools

HOXB7 siRNA Delivered by Hybrid Nanoparticles and the Co-Therapy with Tamoxifen: Promising Strategy against Hormone Receptor-Positive Breast Cancer

“…HOXB7 promotes TAM resistance playing a role in two main pathways: EGFR and ER [10,11]. Thus, overexpression of HOXB7 promotes HOXB7 is correlated with clinical progression, poor outcome of breast cancer patients [10,13] and shorter relapse-free survival [14] Targeted therapies such as RNA interference (RNAi) therapy provide new perspectives for the treatment of several diseases, including breast cancer [15]. This became more tangible with the approval of the first RNAi-based medicine ONPATTRO ® by the USA Food and Drug Administration, 20 years after the elucidation of the RNAi mechanism by Fire and co-workers [16].…”

“…This became more tangible with the approval of the first RNAi-based medicine ONPATTRO ® by the USA Food and Drug Administration, 20 years after the elucidation of the RNAi mechanism by Fire and co-workers [16]. RNAi therapy is based on this mechanism, where small RNA molecules (siRNA) sequence-specifically binds to messenger RNAs (mRNAs), resulting in the cleavage and degradation of the targeted mRNA to inhibit protein synthesis [15].…”