Small Interfering RNA (siRNA) Therapy - StatPearls - NCBI Bookshelf
2024年3月20日 · This activity focuses on small interfering RNA (siRNA) therapy, exemplified by FDA-approved agents such as patisiran, givosiran, lumasiran, inclisiran, nedosiran, and vutrisiran.
Therapeutic siRNA: state of the art | Signal Transduction and …
2020年6月19日 · siRNA has innate advantages over small molecular therapeutics and monoclonal antibody drugs because siRNA executes its function by complete Watson–Crick base pairing with mRNA, whereas small...
siRNA Drugs: Here to Stay - PMC
The core promise of siRNA drugs remains—only the primary nucleotide sequence of the target is required to design a new drug, allowing the possibility for rational drug design that avoids the complexity of high-throughput screening and can, theoretically, target any …
Where should siRNAs go: applicable organs for siRNA drugs
2023年7月10日 · Although all FDA-approved siRNA drugs target the hepatocytes of the liver, siRNA-based drugs targeting different organs are in clinical trials. In this review, we introduce siRNA drugs...
The growth of siRNA-based therapeutics: Updated clinical studies
More than two decades after the natural gene-silencing mechanism of RNA interference was elucidated, small interfering RNA (siRNA)-based therapeutics have finally broken into the pharmaceutical market.
The growth of siRNA-based therapeutics: updated clinical studies
2022年7月2日 · siRNA drug design and modifications can now be rationally designed by use of algorithms. Years of research and testing have yielded substitution and modification predictions that increase stability, protection, and bioavailability while maintaining efficacy and potency.
siRNA: Mechanism of action, challenges, and therapeutic approaches
2021年8月15日 · 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.
Development, Opportunities and Challenges of siRNA Nucleic Acid Drugs
2024年12月21日 · The FDA has approved six siRNA drugs in recent years: Patisiran, Givosiran, Lumasiran, Vutrisiran, Inclisiran, and Nedosiran. siRNA drugs are based on the post-transcriptional gene regulation mechanism of RNA interference.
siRNA Drugs: Here to Stay: Molecular Therapy - Cell Press
2021年2月3日 · The siRNA specifically targets the mutant mRNA in the liver, thereby preventing formation of the misfolded protein and subsequently the deposits. Indeed, the conceptual beauty of siRNA drugs derives from their specific destruction of a …
Unexpected discovery opens doors for improved siRNA therapies
2024年12月20日 · The first siRNA drug, patisiran, was approved by the U.S. Food and Drug Administration (FDA) in 2018 to treat a debilitating genetic disorder called hereditary transthyretin amyloidosis.
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