Strategies for improving the specificity of siRNAs for enhanced therapeutic potential.
Expert Opin Drug Discov
; 13(8): 709-725, 2018 08.
Article
em En
| MEDLINE
| ID: mdl-29902093
ABSTRACT
INTRODUCTION:
RNA interference has become a tool of choice in the development of drugs in various therapeutic areas of Post Transcriptional Gene Silencing (PTGS). The critical element in developing successful RNAi therapeutics lies in designing small interfering RNA (siRNA) using an efficient algorithm satisfying the designing criteria. Further, translation of siRNA from bench-side to bedside needs an efficient delivery system and/or chemical modification. Areas covered This review emphasizes the importance of dicer, the criteria for efficient siRNA design, the currently available algorithms and strategies to overcome off-target effects, immune stimulatory effects and endosomal trap. Expert opinion Specificity and stability are the primary concerns for siRNA therapeutics. The design criteria and algorithms should be chosen rationally to have a siRNA sequence that binds to the corresponding mRNA as it happens in the Watson and Crick base pairing. However, it must evade a few more hurdles (Endocytosis, Serum stability etc.) to be functional in the cytosol.Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
RNA Interferente Pequeno
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Interferência de RNA
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Desenvolvimento de Medicamentos
Idioma:
En
Ano de publicação:
2018
Tipo de documento:
Article