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Clinical pharmacology of siRNA therapeutics: current status and future prospects
Expert Rev Clin Pharmacol. 2022 Oct 17. doi: 10.1080/17512433.2022.2136166. Online ahead of print.ABSTRACTINTRODUCTION: Small interfering RNA (siRNA) has emerged as a powerful tool for post-transcriptional downregulation of multiple genes for various therapies. Naked siRNA molecules are surrounded by several barriers that tackle their optimum delivery to target tissues such as limited cellular uptake, short circulation time, degradation by endonucleases, glomerular filtration, and capturing by the reticuloendothelial system (RES).AREAS COVERED: This review provides insights into studies that investigate various siRNA-based...
Source: Expert Review of Clinical Pharmacology - October 17, 2022 Category: Drugs & Pharmacology Authors: Ahmed Khaled Abosalha Jacqueline Boyajian Waqar Ahmad Paromita Islam Merry Ghebretatios Sabrina Schaly Rahul Thareja Karan Arora Satya Prakash Source Type: research

Therapeutic siRNA: State-of-the-Art and Future Perspectives
This article reviews the knowledge on siRNA design and chemical modification, as well as issues related to siRNA delivery that may be addressed using different delivery systems. Details on the mode of action and clinical status of the various siRNA therapeutics are provided, before giving an outlook on issues regarding the future of siRNA drugs and on their potential as one emerging standard modality in pharmacotherapy. Notably, this may also cover otherwise un-druggable diseases, the definition of non-coding RNAs as targets, and novel concepts of personalized and combination treatment regimens.
Source: BioDrugs - August 23, 2022 Category: Drugs & Pharmacology Source Type: research

Delivery Strategies for mRNA Vaccines
AbstractThe therapeutic potential for messenger RNA (mRNA) in infectious diseases and cancer was first realized almost three decades ago, but only in 2018 did the first lipid nanoparticle-based small interfering RNA (siRNA) therapy reach the market with the United States Food and Drug Administration (FDA) approval of patisiran (Onpattro ™) for hereditary ATTR amyloidosis. This was largely made possible by major advances in the formulation technology for stabilized lipid-based nanoparticles (LNPs). Design of the cationic ionizable lipids, which are a key component of the LNP formulations, with an acid dissociation constan...
Source: Pharmaceutical Medicine - January 30, 2022 Category: Drugs & Pharmacology Source Type: research

RNAi-based therapeutics and tumor targeted delivery in cancer
Adv Drug Deliv Rev. 2022 Jan 18:114113. doi: 10.1016/j.addr.2022.114113. Online ahead of print.ABSTRACTOver the past decade, non-coding RNA-based therapeutics have proven as a great potential for the development of targeted therapies for cancer and other diseases. The discovery of the critical function of microRNAs (miRNAs) has generated great excitement in developing miRNA-based therapies. The dysregulation of miRNAs contributes to the pathogenesis of various human diseases and cancers by modulating genes that are involved in critical cellular processes, including cell proliferation, differentiation, apoptosis, angiogenes...
Source: Advanced Drug Delivery Reviews - January 22, 2022 Category: Drugs & Pharmacology Authors: Goknur Kara George A Calin Bulent Ozpolat Source Type: research

Givosiran to treat acute porphyria.
Authors: Honor A, Rudnick SR, Bonkovsky HL Abstract Porphyrias are a family of rare diseases chiefly due to inborn errors of heme biosynthesis. The porphyrias are generally characterized either by the main site of overproduction of heme precursors (hepatic or erythropoietic) or the main clinical manifestations (acute or cutaneous). The regulation of 5- (or δ)-aminolevulinic acid synthase 1 (ALAS1) plays a key role in the pathway of normal hepatic heme synthesis, providing insight into the pathophysiologic mechanisms and potential therapeutic targets for the treatment of the porphyrias. Givosiran (Givlaari; Alnylam...
Source: Drugs of Today - February 18, 2021 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research

Small Interfering RNA Therapeutic Inclisiran: A New Approach to Targeting PCSK9
AbstractHypercholesterolemia is a leading cause of cardiovascular disease and mortality in men and women throughout the USA and abroad. The development of statins (HMG-CoA reductase inhibitors) to lower plasma atherogenic cholesterol levels and improve cardiovascular outcomes represents one of the greatest contributions to clinical science in the twentieth century, although residual risk remains even among statin-treated patients. Our understanding of lipid metabolism took a giant leap forward in 2003 with the discovery of proprotein convertase subtilsin/kexin type  9 (PCSK9), a low abundance circulating protein with an o...
Source: BioDrugs - November 27, 2019 Category: Drugs & Pharmacology Source Type: research

Patisiran for the treatment of patients with familial amyloid polyneuropathy.
Authors: Rizk M, Tüzmen S Abstract Onpattro, also commonly known as patisiran, is a small interfering RNA (siRNA) molecule packaged within a lipid nanoparticle and is transported into the cell to target transthyretin gene (TTR) messenger mRNA (mRNA) by attaching to its complementary sequence. The target mRNA is degraded and both mutant and wild-type amyloid transthyretin (ATTR) protein production becomes suppressed. This drug was developed by Alnylam Pharmaceuticals to treat a rare disease called hereditary ATTR (hATTR) amyloidosis. This disease develops as a result of the deposition of toxic aggregates of misfold...
Source: Drugs of Today - May 28, 2019 Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research