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Rna Interference-Based Therapies For The Reduction Of

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Introduction RNA interference (RNAi) therapy represents an evolving advancement in the management of dyslipidemia. One prominent form of RNAi therapy is small interfering RNA (siRNA), which has emerged as a

This review explores the current state and prospects of RNA-based therapies in treating HCC, highlighting the advances, challenges, and potential of this emerging field, and providing new directions for designing precise, effective, and personalized RNA nanomedicines.

RNA Interference (RNAi): A Novel Approach In Fighting Disease

This review focuses on antisense oligonucleotides and small interfering ribonucleic acid therapies approved or under development for the management of lipid disorders. Recent advances in RNA-based therapeutics allow tissue-specific targeting improving This activity focuses on small interfering RNA (siRNA) therapy, exemplified by FDA-approved agents such as patisiran, givosiran, lumasiran, inclisiran, nedosiran, and vutrisiran. These therapies are pivotal in managing rare metabolic ailments, including hereditary transthyretin amyloidosis (hATTR), acute hepatic porphyria (AHP), primary hyperoxaluria type 1 (PH1), and RNA interference (RNAi) therapies represent a revolutionary paradigm in medicine, offering innovative ways to manipulate gene expression for therapeutic purposes. The ability to selectively silence or downregulate specific genes has opened new avenues for treating a myriad of diseases. 1 Understanding RNA Interference RNAi is a natural cellular process that

Gene silencing by RNA interference: a review

Introduction: RNA interference (RNAi) therapy represents an evolving advancement in the management of dyslipidemia. One prominent form of RNAi therapy is small interfering RNA therapeutics allow tissue specific targeting (siRNA), which has emerged as a promising therapeutic strategy. This study aims to critically analyze the efficacy and safety of siRNA in the treatment of dyslipidemia.

RNA interference (RNAi) therapy is a rapidly emerging platform for personalized cancer treatment. Recent advances in small interfering RNA delivery and target selection provide unprecedented opportunities for clinical translation. Here, we discuss these advances and present strategies for making RNAi-based therapy a viable part of cancer Findings The first RNAi based management. The urgent need to assess the effectiveness, safety, and translational potential of these novel therapies for heart failure is the reason for doing a comprehensive review on RNA-based medicines. RNA-based therapeutics provide a fresh approach by focusing on the underlying genetic mechanisms driving the course of the disease.

RNA interference (RNAi) is a collection of small RNA directed mechanisms that result in sequence specific inhibition of gene expression. The notion that RNAi could lead to a new class of therapeutics caught the attention of many investigators soon The recent approval of the first RNA interference (RNAi)-based therapy has generated considerable excitement in the field. Here, Rossi and colleagues hepatocyte uptake discuss key advances in the design and RNA interference, using siRNAs, is a novel therapeutic strategy that has recently been translated from bench to bedside, and gradually evolved from use in rare genetic disorders to common chronic conditions with the licensing of inclisiran for the treatment of hypercholesterolemia by the US Food and Drug Administration in 2021. Zilebesiran is a novel

RNA-targeting therapies hold promise for contributing to HBV cure regimens, but challenges remain before that promise can be realized. RNA interference (RNAi)-based gene silencing has emerged as a potent method for regulating gene expression, with applications spanning biology, medicine, and biotechnology. RNAi exploits natural cellular in modern machinery to selectively suppress target gene expression through the targeted degradation of mRNA based on its specific sequence. MicroRNAs (miRNAs) and However, ldl-c levels reduction may not eliminate the risk of significant cardiovascular events.中文翻译： 基于 RNA 干扰的疗法可降低心血管风险 * 在全球范围内，心血管疾病的发病率仍然稳步上升，从 1990 年的 2.71 亿增加到 2019 年的 5.23 亿。

Nucleic acid-based therapeutics represent a groundbreaking frontier in modern medicine, utilizing the precise targeting capabilities of synthetic or naturally occurring DNA and RNA molecules to modulate gene expression. These therapies, also referred to as oligonucleotide therapeutics or gene-silencing technologies, interact directly with specific RNA or DNA

Lowering low‐density lipoprotein cholesterol (LDL‐C) is a cornerstone of reducing risk for atherosclerotic cardiovascular disease. Despite the approval of nonstatin therapies for LDL‐C the past 2 lowering over the past 2 decades, these medications are underused, and most patients are still not at guideline‐recommended LDL‐C goals. Barriers include poor adherence, clinical

RNAi Therapies: Drugging the Undruggable

In contrast, RNA interfering (RNAi)-based therapies utilize double-stranded RNA and interfere with mRNA expression. The majority of RNAi-based therapies employ noncoding small interfering RNAs Key Points RNA interference (RNAi) is a powerful approach for reducing expression of endogenously expressed proteins for biological applications, or targeting the expression of pathological proteins for therapy. Several delivery methods are 心血管疾病的发病率仍然稳步上升 从 available to achieve RNAi in ex vivo and in vivo settings for therapeutic results. The development of RNAi-based therapeutics BACKGROUND: Apolipoprotein C-III (APOC3) inhibits triglyceride clearance by reducing lipoprotein lipase–mediated hydrolysis and hepatocyte uptake of triglyceride-rich lipoproteins. ARO-APOC3, a hepatocyte-targeting RNA interference therapeutic, inhibits APOC3 messenger ribonucleic acid expression, l

RNA-targeted therapy can precisely modulate the function of target RNA with minimal off-target effects and can be rationally designed based on sequence data. ASOs and siRNA-based drugs have unique capabilities for using in target groups of patients or can be tailored as patient-customized N-of-1 therapeutic approach.

  • RNA-based therapy in the management of lipid disorders: a review
  • RNA interference approaches for treatment of HIV-1 infection
  • RNA interference in the era of nucleic acid therapeutics
  • RNA interference: A futuristic tool and its therapeutic applications

Three randomized clinical trials presented at ACC.24 demonstrate that olezarsen and plozasiran, RNA-based therapies that target APOC3, can robustly reduce plasma triglyceride levels in patients Purpose of Review Current treatments for chronic hepatitis B (CHB) are associated with low rates of cure. Functional cure has been accepted as a viable treatment end point in CHB. There have been substantial advances in the field of RNA interference (RNAi) therapeutics across a wide range of specialties, and the clinical pipeline now encompasses Instead, this review describes the major families of RNA therapies, summarizes the RNA therapeutics currently in use (or in development) for cardiovascular indications (Table 1), and describes the mechanism of action for selected RNA drugs (Figure 1).

For a given reduction in apoB levels, genetically predicted reductions in PCSK9 function (mimicking PCSK9 neutralizing antibodies) and liver PCSK9 gene expression levels (mimicking PCSK9 RNA interference) were comparably associated with a lower risk of coronary artery disease. These genetic data sug RNA interference is a novel treatment strategy for chronic hepatitis B. RNA interference agents have entered phase II/III clinical trials, demonstrating promising results in inducing hepatitis B surface antigen seroclearance. RNA interference agents will likely be the keystone of future hepatitis B treatment.

However, some aspects associated with the use of RNA-based drugs still need to be improved, like the need for increased stability, the reduction in off-target effects, and the advent of flulike symptoms. The RNA interference (RNAi) drug ARC-520 was shown to be effective in reducing serum hepatitis B virus (HBV) DNA, hepatitis B e antigen (HBeAg) and hepatitis B surface antigen (HBsAg) in HBeAg

Abstract The discovery of RNA interference in 1998 opened avenues for the manipulation of gene expression, leading to the development of small interfering RNA (siRNA) drugs. Pa-tisiran, the first FDA-approved siRNA medication, targets hereditary transthyretin amyloidosis with polyneuropathy. Givosiran, lumasiran and nedosiran further expand siRNA applications in RNAi, particularly using small interfering RNA (siRNA), targets viral RNA that can be used to inhibit hepatitis B virus (HBV) replication. Several candidates are currently being studied and have exhibited varying success in reducing hepatitis B surface antigen (HBsAg) levels, with some showing sustained HBsAg loss after cessation of therapy. RNA has emerged as a revolutionary and important tool in the battle against emerging infectious diseases, with roles extending beyond its applications in vaccines, in which it is used in the response to the COVID-19 pandemic. Since their development in the 1990s, RNA interference (RNAi) therapeutics have demonstrated potential in reducing the expression of

In this review, we discuss and describe novel RNA interference (RNAi)-based therapies in clinical trials and on the market. Recent Findings The first RNAi-based therapies have entered clinical use for the control of atherosclerosis risk factors, i.e., blood cholesterol levels.

Since the first RNA therapy drug was approved for the treatment of cytomegalovirus retinitis in 1998 [10], the Food and Drug Administration has sanctioned 9 ASOs [11], 5 siRNAs [12], and 2 mRNA-based [13] therapeutics for clinical use, demonstrating the utility of nucleic acid–based therapies.

Conclusion: The newer RNA interference (RNAi) therapies appear safe and have excellent TG-lowering efficacy in patients with HTG and MHL. Each therapy targets specific molecules while also significantly impacting other lipid parameters. The promising results of these trials indicate potential improvements in lipid therapy and cardiovascular risk reduction, with ongoing studies expected to further refine the role of the novel RNA-based agents in effective lipid management.

Abstract Purpose of Review Atherosclerosis, defined by inflammation and accumulation of cholesterol, extracellular matrix, and cell debris into the arteries is a common factor behind cardiovascular diseases (CVD), such as coronary artery disease, peripheral artery disease, and stroke. In this review, we discuss and describe novel RNA interference (RNAi)-based therapies This review will cover the mechanisms of RNA therapy design, current FDA-approved RNA therapies, RNA therapies in clinical trials, and RNAs with clinical potential for treating patients suffering from various conditions.

No meta-analysis has holistically analyzed and summarized the safety and therapeutic efficacy of the newer RNA interference (RNAi) therapies, olezarsen, plozasiran, and zodasiran, in managing conditions associated with hypertriglyceridemia (HTG).