Abstract
Retinal gene transfer holds big promises for the treatment of inherited and non-inherited blinding diseases, such as retinitis pigmentosa or age-related macular degeneration. Key to the development of successful gene-based therapies for the eye are efficient tools for retinal gene transfer. Vectors based on adeno-associated viruses (AAV) are able to transduce robustly and persistently different retinal cell types of animal models after a single intraocular administration. Recombinant AAV (rAAV) vectors are versatile gene transfer tools in that capsid proteins from dozens of AAV serotypes can be easily interchanged, resulting in the creation of recombinant vectors with unique transduction properties. This has allowed successful proof-of-principle studies using rAAV-mediated gene transfer to restore retinal morphology and function in small and large animal models of retinal diseases. In addition, gene delivery using rAAV vectors in the eye seems to have appropriate biosafety characteristics to rapidly move it from bench to bedside. All the above aspects will be reviewed and discussed in detail below.
Keywords: retina, gene transfer, raav, retinal degeneration, retinitis pigmentosa, neovascular diseases
Current Gene Therapy
Title: Adeno-Associated Viral Vectors for Retinal Gene Transfer and Treatment of Retinal Diseases
Volume: 5 Issue: 3
Author(s): Alberto Auricchio and Fabienne Rolling
Affiliation:
Keywords: retina, gene transfer, raav, retinal degeneration, retinitis pigmentosa, neovascular diseases
Abstract: Retinal gene transfer holds big promises for the treatment of inherited and non-inherited blinding diseases, such as retinitis pigmentosa or age-related macular degeneration. Key to the development of successful gene-based therapies for the eye are efficient tools for retinal gene transfer. Vectors based on adeno-associated viruses (AAV) are able to transduce robustly and persistently different retinal cell types of animal models after a single intraocular administration. Recombinant AAV (rAAV) vectors are versatile gene transfer tools in that capsid proteins from dozens of AAV serotypes can be easily interchanged, resulting in the creation of recombinant vectors with unique transduction properties. This has allowed successful proof-of-principle studies using rAAV-mediated gene transfer to restore retinal morphology and function in small and large animal models of retinal diseases. In addition, gene delivery using rAAV vectors in the eye seems to have appropriate biosafety characteristics to rapidly move it from bench to bedside. All the above aspects will be reviewed and discussed in detail below.
Export Options
About this article
Cite this article as:
Auricchio Alberto and Rolling Fabienne, Adeno-Associated Viral Vectors for Retinal Gene Transfer and Treatment of Retinal Diseases, Current Gene Therapy 2005; 5 (3) . https://dx.doi.org/10.2174/1566523054065020
DOI https://dx.doi.org/10.2174/1566523054065020 |
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
-
Development and Clinical use of Prasugrel and Ticagrelor
Current Pharmaceutical Design Sudden Infant Death Syndrome from Epidemiology to Pathophysiology
Current Pediatric Reviews Atrial Macroreentry in Congenital Heart Disease
Current Cardiology Reviews Nursing Assessment, Education, and Care of Extremely Premature Neonates with Patent Ductus Arteriosus
Current Pediatric Reviews Premature Adrenarche and its Association with Cardiovascular Risk in Females
Current Pharmaceutical Design Pharmacogenetically Tailored Treatments for Heart Disease
Current Pharmaceutical Design Varicella-Zoster Virus Infections During Pregnancy: Epidemiology, Clinical Symptoms, Diagnosis, Prevention and Therapy
Current Pediatric Reviews Current Management of Neonatal Liver Tumors
Current Pediatric Reviews Cardiovascular Involvement in Pediatric Systemic Autoimmune Diseases: The Emerging Role of Noninvasive Cardiovascular Imaging
Inflammation & Allergy - Drug Targets (Discontinued) The Role of Beta-Blocker in Heart Failure in Adults with Congenital Heart Disease
Reviews on Recent Clinical Trials The Fetal Cardiac Function
Current Cardiology Reviews Pharmacologic Treatment of Pulmonary Arterial Hypertension
Current Pharmaceutical Design Pharmacotherapy in Neonatal and Pediatric Extracorporeal Membrane Oxygenation (ECMO)
Current Drug Metabolism Novel Therapeutic Agents in the Management of Hemorrhage and Thrombosis
Cardiovascular & Hematological Agents in Medicinal Chemistry Peripheral Chemoreceptors and Sudden Infant Death Syndrome: A Wide Open Problem
Current Cardiology Reviews Psychoemotional Background of Temporomandibular Joint Dysfunction and Possible Drug Therapy
Letters in Drug Design & Discovery A Summary of Electrospun Nanofibers as Drug Delivery System: Drugs Loaded and Biopolymers Used as Matrices
Current Drug Delivery Extending Life Using Tissue and Organ Replacement
Current Aging Science Genetic Basis of Ureterocele
Current Genomics Economic Evaluation in Paediatric Practice: Examples from Cardiac Critical Care
Current Pediatric Reviews