Abstract
RNA interference has emerged as an innovative technology for gene silencing that degrades mRNAs complementary to the antisense strands of double-stranded, short interfering RNAs (siRNAs). Its therapeutic application has important advantages over small-molecule drugs since offers the possibility of targeting virtually all genes and allows selective silencing of one or several genes. So far, a relative small proportion of cellular proteins can bind and respond to chemical drugs. Based on that, RNA interference-mediated gene silencing is widely considered as a crucial breakthrough in molecular biology with a direct translation to medicine. The liver has been widely chosen as a model system for the development of RNA interference therapy due to the convenience and availability of effective delivery into this tissue. Numerous preclinical models have revealed promising results, but the safety of this technology remains the primary challenge in developing siRNA based treatments. Liver diseases comprise a broad spectrum of genetic and non-genetic pathologies including acute fulminant liver injury that demands urgent medical care, or chronic pathologies such as nonalcoholic fatty liver (NAFLD), alcoholic liver disease, liver cirrhosis, viral hepatitis and hepatocellular carcinoma (HCC). In some cases restoration of liver function is not possible and alternatives to liver transplantation offering novel and efficient therapeutic approaches are urgently needed. In this review, we describe recent insights on the advantages of using RNA interference in preclinical settings as a targeted strategy with potential to markedly improve the treatment of liver diseases.
Keywords: RNA interference, liver, acute liver failure, liver metabolism, fibrosis, hepatocellular carcinoma.
Current Pharmaceutical Design
Title:RNA Interference as a Therapeutic Strategy for the Treatment of Liver Diseases
Volume: 21 Issue: 31
Author(s): Agueda Gonzalez-Rodriguez and Angela M. Valverde
Affiliation:
Keywords: RNA interference, liver, acute liver failure, liver metabolism, fibrosis, hepatocellular carcinoma.
Abstract: RNA interference has emerged as an innovative technology for gene silencing that degrades mRNAs complementary to the antisense strands of double-stranded, short interfering RNAs (siRNAs). Its therapeutic application has important advantages over small-molecule drugs since offers the possibility of targeting virtually all genes and allows selective silencing of one or several genes. So far, a relative small proportion of cellular proteins can bind and respond to chemical drugs. Based on that, RNA interference-mediated gene silencing is widely considered as a crucial breakthrough in molecular biology with a direct translation to medicine. The liver has been widely chosen as a model system for the development of RNA interference therapy due to the convenience and availability of effective delivery into this tissue. Numerous preclinical models have revealed promising results, but the safety of this technology remains the primary challenge in developing siRNA based treatments. Liver diseases comprise a broad spectrum of genetic and non-genetic pathologies including acute fulminant liver injury that demands urgent medical care, or chronic pathologies such as nonalcoholic fatty liver (NAFLD), alcoholic liver disease, liver cirrhosis, viral hepatitis and hepatocellular carcinoma (HCC). In some cases restoration of liver function is not possible and alternatives to liver transplantation offering novel and efficient therapeutic approaches are urgently needed. In this review, we describe recent insights on the advantages of using RNA interference in preclinical settings as a targeted strategy with potential to markedly improve the treatment of liver diseases.
Export Options
About this article
Cite this article as:
Gonzalez-Rodriguez Agueda and M. Valverde Angela, RNA Interference as a Therapeutic Strategy for the Treatment of Liver Diseases, Current Pharmaceutical Design 2015; 21 (31) . https://dx.doi.org/10.2174/138161282131151013190740
DOI https://dx.doi.org/10.2174/138161282131151013190740 |
Print ISSN 1381-6128 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-4286 |
Call for Papers in Thematic Issues
"Tuberculosis Prevention, Diagnosis and Drug Discovery"
The Nobel Prize-winning discoveries of Mycobacterium tuberculosis and streptomycin have enabled an appropriate diagnosis and an effective treatment of tuberculosis (TB). Since then, many newer diagnosis methods and drugs have been saving millions of lives. Despite advances in the past, TB is still a leading cause of infectious disease mortality ...read more
Current Pharmaceutical challenges in the treatment and diagnosis of neurological dysfunctions
Neurological dysfunctions (MND, ALS, MS, PD, AD, HD, ALS, Autism, OCD etc..) present significant challenges in both diagnosis and treatment, often necessitating innovative approaches and therapeutic interventions. This thematic issue aims to explore the current pharmaceutical landscape surrounding neurological disorders, shedding light on the challenges faced by researchers, clinicians, and ...read more
Emerging and re-emerging diseases
Faced with a possible endemic situation of COVID-19, the world has experienced two important phenomena, the emergence of new infectious diseases and/or the resurgence of previously eradicated infectious diseases. Furthermore, the geographic distribution of such diseases has also undergone changes. This context, in turn, may have a strong relationship with ...read more
Melanoma and Non-Melanoma Skin Cancer Treatment: Standard of Care and Recent Advances
In this thematic issue, we aim to provide a standard of care of the diagnosis and treatment of melanoma and non-melanoma skin cancer. The editor will invite authors from different countries who will write review articles of melanoma and non-melanoma skin cancers. The Diagnosis, Staging, Surgical Treatment, Non-Surgical Treatment all ...read more
- Author Guidelines
- Graphical Abstracts
- Fabricating and Stating False Information
- Research Misconduct
- Post Publication Discussions and Corrections
- Publishing Ethics and Rectitude
- Increase Visibility of Your Article
- Archiving Policies
- Peer Review Workflow
- Order Your Article Before Print
- Promote Your Article
- Manuscript Transfer Facility
- Editorial Policies
- Allegations from Whistleblowers
- Announcements
Related Articles
-
Implications of the Dominant Role of Transporters in Drug Uptake by Cells
Current Topics in Medicinal Chemistry Isovitexin Inhibits Stemness and Induces Apoptosis in Hepatocellular Carcinoma SK-Hep-1 Spheroids by Upregulating miR-34a Expression
Anti-Cancer Agents in Medicinal Chemistry Novel Approaches to Modulate Apoptosis Resistance: Basic and Clinical Implications in the Treatment of Chronic Lymphocytic Leukemia (CLL)
Current Drug Delivery Chalcone Derivatives Activate and Desensitize the Transient Receptor Potential Ankyrin 1 Cation Channel, Subfamily A, Member 1 TRPA1 Ion Channel: Structure-Activity Relationships in vitro and Anti-Nociceptive and Anti-inflammatory Activity in vivo
CNS & Neurological Disorders - Drug Targets Novel Immunotherapeutic Strategies for Invasive Fungal Disease
Current Drug Targets - Cardiovascular & Hematological Disorders Synthesis and Antiproliferative Assay of Norcantharidin Derivatives in Cancer Cells
Medicinal Chemistry Recent Developments of Rebeccamycin Analogues as Topoisomerase I Inhibitors and Antitumor Agents.
Current Medicinal Chemistry Prognostic and Predictive Biomarkers in Cancer
Current Cancer Drug Targets Alternative Splicing in Chronic Myeloid Leukemia (CML): A Novel Therapeutic Target?
Current Cancer Drug Targets Bench to Bedside Targeting of FLT3 in Acute Leukemia
Current Drug Targets Polymeric Carrier Systems for siRNA Delivery
Current Topics in Medicinal Chemistry Role of α7-Nicotinic Acetylcholine Receptor in Normal and Cancer Stem Cells
Current Drug Targets Pharmacophore Modelling as a Virtual Screening Tool for the Discovery of Small Molecule Protein-protein Interaction Inhibitors
Current Pharmaceutical Design The Inverse Relationship Between Cancer and Alzheimer's Disease: A Possible Mechanism
Current Alzheimer Research QRPR and HCQRPQ, Two Peptides from Soybean, have an Inhibitory Effect on the Proliferation of HepG2 Cells
Protein & Peptide Letters Nucleic Acid-Based Aptamers: Applications, Development and Clinical Trials
Current Medicinal Chemistry Targeting Multiplicity: The Key Factor for Anti-Cancer Nanoparticles
Current Medicinal Chemistry The Role of the Tyrosine Kinase Inhibitor STI571 in the Treatment of Cancer
Current Cancer Drug Targets Targeted Therapies in Hepatocellular Carcinoma
Current Medicinal Chemistry Targeted – Therapy and Imaging Response: A New Paradigm For Clinical Evaluation?
Reviews on Recent Clinical Trials