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Total 35 results found since Jan 2013.

Where should siRNAs go: applicable organs for siRNA drugs
Experimental & Molecular Medicine, Published online: 10 July 2023; doi:10.1038/s12276-023-00998-yRefining the design and delivery of RNA-based drugs could improve the chances of targeting diseases in complex organs. Gene expression is regulated by small interfering RNAs (siRNAs), which bind to messenger RNA sequences, preventing subsequent gene expression. Drugs based on siRNA show promise as safe and specialised for multiple diseases. Jinju Han and co-workers at the Korea Advanced Institute for Science and Technology in Daejeon, South Korea, reviewed the current status of siRNA drugs. Five siRNAs approved by the US Fo...
Source: Experimental and Molecular Medicine - July 10, 2023 Category: Molecular Biology Authors: Insook Ahn Chanhee S. Kang Jinju Han Source Type: research

DKK1-targeting cholesterol-modified siRNA implication in hair growth regulation
In this report, we have selected a small interfering RNA (siRNA) targeting DKK1 in vitro via qPCR and then tested its efficacy in vivo on the depilated dorsal skin of the mice. The changes in hair growth in different groups were observed over time. Moreover, the visual observation of the hair growth and hematoxylin and eosin (HE) staining showed that DKK1-targeting siRNA reveals non-inferior results compared with the mice treated with the Food and Drug Administration (FDA)-approved, commercially available minoxidil (5%) topical solution that was used as a positive control. Both- positive control and DKK1-targeting siRNA gr...
Source: Biochemical and Biophysical Research communications - May 27, 2023 Category: Biochemistry Authors: Dimitri Papukashvili Cong Liu Nino Rcheulishvili Fengfei Xie Xingyun Wang Shunping Feng Xiu Sun Chi Zhang Yingyu Li Yunjiao He Peng George Wang Source Type: research

Viruses, Vol. 15, Pages 105: Topoisomerase II as a Novel Antiviral Target against Panarenaviral Diseases
ro Yasuda Although many arenaviruses cause severe diseases with high fatality rates each year, treatment options are limited to off-label use of ribavirin, and a Food and Drug Administration (FDA)-approved vaccine is not available. To identify novel therapeutic candidates against arenaviral diseases, an RNA polymerase I-driven minigenome (MG) expression system for Lassa virus (LASV) was developed and optimized for high-throughput screening (HTS). Using this system, we screened 2595 FDA-approved compounds for inhibitors of LASV genome replication and identified multiple compounds including pixantrone maleate, a topoisom...
Source: Viruses - December 30, 2022 Category: Virology Authors: Tosin Oladipo Afowowe Yasuteru Sakurai Shuzo Urata Vahid Rajabali Zadeh Jiro Yasuda Tags: Article Source Type: research

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

Cancers, Vol. 14, Pages 3162: Knockdown of 15-bp Deletion-Type v-raf Murine Sarcoma Viral Oncogene Homolog B1 mRNA in Pancreatic Ductal Adenocarcinoma Cells Repressed Cell Growth In Vitro and Tumor Volume In Vivo
In this study, siRNA was designed for the targeted knockdown of 15-bp deletion-type BRAF mRNA. This siRNA repressed the phosphorylation of extracellular-signal-regulated kinase proteins downstream of BRAF and suppressed cell growth in vitro and in vivo. Furthermore, siRNAs with 2′-O-methyl modifications at positions 2–5 reduce the seed-dependent off-target effects, as confirmed by reporter and microarray analyses. Thus, such siRNA is a promising candidate therapy for 15-bp deletion-type BRAF-induced tumorigenesis.
Source: Cancers - June 28, 2022 Category: Cancer & Oncology Authors: Jiaxuan Song Yoshiaki Kobayashi Yoshimasa Asano Atsushi Sato Hiroaki Taniguchi Kumiko Ui-Tei Tags: Article Source Type: research

New Strategies for Lowering Low-Density Lipoprotein Cholesterol for Cardiovascular Disease Prevention
AbstractPurpose of ReviewThe primary and secondary prevention of atherosclerotic cardiovascular disease (ASCVD) relies on optimizing cardiovascular health and appropriate pharmacotherapy, a mainstay of which is low-density lipoprotein cholesterol (LDL-C) lowering. Typically, statin therapy remains the first-line approach. Advances in technology and understanding of lipid metabolism have facilitated the development of several novel therapeutic targets and medications within the last decade. This review focuses on medications recently approved by the US Food and Drug Administration (FDA) for the reduction of LDL-C and ASCVD ...
Source: Current Cardiovascular Risk Reports - June 25, 2022 Category: Cardiology 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