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Eur J Pharmacol ; 905: 174178, 2021 Aug 15.
Artigo em Inglês | MEDLINE | ID: mdl-34044011

RESUMO

Owing to specific and compelling gene silencing, RNA interference (RNAi) is expected to become an essential approach in treating a variety of infectious, hemato-oncological, cardiovascular, and neurodegenerative conditions. The mechanism of action of small interfering RNA (siRNA) is based on post-transcriptional gene silencing. siRNA molecules are usually specific and efficient in the knockdown of disease-related genes. However, they are characterized by low cellular uptake and are susceptible to nuclease-mediated degradation. Therefore, siRNAs require a carrier for their protection and efficient delivery into target cells. The current review highlights the siRNA-based mechanism of action, challanges, and recent advances in clinical applications.


Assuntos
RNA Interferente Pequeno/farmacologia , RNA Interferente Pequeno/uso terapêutico , Animais , Ensaios Clínicos como Assunto , Sistemas de Liberação de Medicamentos , Inativação Gênica , Marcação de Genes , Humanos , Interferência de RNA , RNA Interferente Pequeno/química
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