Abstract
Despite the fact that adeno-associated virus type 2 (AAV2) is an extremely attractive gene therapy vector, its application has been limited to certain tissues such as muscle and the brain. In an attempt to broaden the array of target organs for this vector, molecular studies on the mechanism(s) of AAV transduction have expanded over the past several years. These studies have led to the development of innovative strategies capable of overcoming intracellular barriers to AAV2 transduction. The basis of these technologic breakthroughs has stemmed from a better understanding of the molecular processes that control AAV entry and intracellular trafficking to the nucleus. This review will focus on the identification of molecular components important for recombinant AAV (rAAV) transduction while highlighting the techniques used to discover them and potential clinical application of research findings.
Keywords: AAV Transduction, Human Gene Therapy, Recombinant AAV (rAA V), Viral Binding, Heparan sulfate proteoglycan, Nuclear Transport, Viral Uncoating, Nuclear Gene Conversion
Current Gene Therapy
Title: Rate Limiting Steps of AAV Transduction and Implications for Human Gene Therapy
Volume: 1 Issue: 2
Author(s): S. Sanlioglu, M. M. Monick, G. Luleci, G. W. Hunninghake and J. F. Engelhardt
Affiliation:
Keywords: AAV Transduction, Human Gene Therapy, Recombinant AAV (rAA V), Viral Binding, Heparan sulfate proteoglycan, Nuclear Transport, Viral Uncoating, Nuclear Gene Conversion
Abstract: Despite the fact that adeno-associated virus type 2 (AAV2) is an extremely attractive gene therapy vector, its application has been limited to certain tissues such as muscle and the brain. In an attempt to broaden the array of target organs for this vector, molecular studies on the mechanism(s) of AAV transduction have expanded over the past several years. These studies have led to the development of innovative strategies capable of overcoming intracellular barriers to AAV2 transduction. The basis of these technologic breakthroughs has stemmed from a better understanding of the molecular processes that control AAV entry and intracellular trafficking to the nucleus. This review will focus on the identification of molecular components important for recombinant AAV (rAAV) transduction while highlighting the techniques used to discover them and potential clinical application of research findings.
Export Options
About this article
Cite this article as:
Sanlioglu S., Monick M. M., Luleci G., Hunninghake W. G. and Engelhardt F. J., Rate Limiting Steps of AAV Transduction and Implications for Human Gene Therapy, Current Gene Therapy 2001; 1 (2) . https://dx.doi.org/10.2174/1566523013348788
DOI https://dx.doi.org/10.2174/1566523013348788 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
Call for Papers in Thematic Issues
Programmed Cell Death Genes in Oncology: Pioneering Therapeutic and Diagnostic Frontiers (BMS-CGT-2024-HT-45)
Programmed Cell Death (PCD) is recognized as a pivotal biological mechanism with far-reaching effects in the realm of cancer therapy. This complex process encompasses a variety of cell death modalities, including apoptosis, autophagic cell death, pyroptosis, and ferroptosis, each of which contributes to the intricate landscape of cancer development and ...read more
Related Journals
- 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
-
New Biomarkers for Colon Cancer Diagnosis: A Review of Recent Patents
Recent Patents on Biomarkers NK-1 Receptor Antagonists: A New Generation of Anticancer Drugs
Mini-Reviews in Medicinal Chemistry The Use of Herbal Medicine in Cancer-related Anorexia/ Cachexia Treatment Around the World
Current Pharmaceutical Design β-Lactams as Neuroprotective Agents
Anti-Infective Agents in Medicinal Chemistry Pathophysiological Role of Mitochondrial Potassium Channels and their Modulation by Drugs
Current Medicinal Chemistry Role of mTOR Signaling in Tumor Cell Motility, Invasion and Metastasis
Current Protein & Peptide Science MDM2 Increases Drug Resistance in Cancer Cells by Inducing EMT Independent of p53
Current Medicinal Chemistry Antagonists of IAP Proteins: Novel Anti-Tumor Agents
Current Medicinal Chemistry Nanocarriers for Effective Brain Drug Delivery
Current Topics in Medicinal Chemistry In Silico Prediction and Validation of Oxygen-Regulated Protein N-myc Downstream Regulated Gene 3 and Virtual Screening of Competitive Inhibitors of L-Lactate as Therapeutics
Letters in Drug Design & Discovery The Monoaminergic Tripartite Synapse: A Putative Target for Currently Available Antidepressant Drugs
Current Drug Targets Protein Secretome Analysis of Evolving and Responding Tumor Ecosystems
Current Proteomics Targeting Epigenetics in Nervous System Disease
CNS & Neurological Disorders - Drug Targets Aldehyde Dehydrogenase as a Marker for Stem Cells
Current Stem Cell Research & Therapy Expression of Specificity Protein Transcription Factors in Pancreatic Cancer and their Association in Prognosis and Therapy
Current Medicinal Chemistry The Impact of Small Heat Shock Proteins (HspBs) in Alzheimer’s and Other Neurological Diseases
Current Pharmaceutical Design Basic Mechanisms Involved in the Anti-Cancer Effects of Melatonin
Current Medicinal Chemistry Second Generation Adeno-Associated Virus Type 2-based Gene Therapy Systems with the Potential for Preferential Integration into AAVS1
Current Gene Therapy The GABAergic System and the Gastrointestinal Physiopathology
Current Pharmaceutical Design Genetic and Epigenetic Drug Targets in Myelodysplastic Syndromes
Current Pharmaceutical Design