Abstract
Ex vivo gene therapy, a technique where genetic manipulation of cells is undertaken remotely and more safely since it is outside the body, is an emerging therapeutic strategy particularly well suited to targeting a specific organ rather than for treating a whole organism. The eye and visual pathways therefore make an attractive target for this approach. With blindness still so prevalent worldwide, new approaches to treatment would also be widely applicable and a significant advance in improving quality of life. Despite being a relatively new approach, ex vivo gene therapy has already achieved significant advances in the treatment of blindness in pre-clinical trials. In particular, advances are being achieved in corneal disease, glaucoma, retinal degeneration, stroke and multiple sclerosis through genetic re-programming of cells to replace degenerate cells and through more refined neuroprotection, modulation of inflammation and replacement of deficient protein. In this review we discuss the latest developments in ex vivo gene therapy relevant to the visual pathways and highlight the challenges that need to be overcome for progress into clinical trials.
Keywords: Ex vivo gene therapy, eye, brain
Current Gene Therapy
Title:Ex Vivo Gene Therapy and Vision
Volume: 12 Issue: 2
Author(s): Kevin Gregory-Evans, A. M.A. Emran Bashar and Malcolm Tan
Affiliation:
Keywords: Ex vivo gene therapy, eye, brain
Abstract: Ex vivo gene therapy, a technique where genetic manipulation of cells is undertaken remotely and more safely since it is outside the body, is an emerging therapeutic strategy particularly well suited to targeting a specific organ rather than for treating a whole organism. The eye and visual pathways therefore make an attractive target for this approach. With blindness still so prevalent worldwide, new approaches to treatment would also be widely applicable and a significant advance in improving quality of life. Despite being a relatively new approach, ex vivo gene therapy has already achieved significant advances in the treatment of blindness in pre-clinical trials. In particular, advances are being achieved in corneal disease, glaucoma, retinal degeneration, stroke and multiple sclerosis through genetic re-programming of cells to replace degenerate cells and through more refined neuroprotection, modulation of inflammation and replacement of deficient protein. In this review we discuss the latest developments in ex vivo gene therapy relevant to the visual pathways and highlight the challenges that need to be overcome for progress into clinical trials.
Export Options
About this article
Cite this article as:
Gregory-Evans Kevin, M.A. Emran Bashar A. and Tan Malcolm, Ex Vivo Gene Therapy and Vision, Current Gene Therapy 2012; 12 (2) . https://dx.doi.org/10.2174/156652312800099607
DOI https://dx.doi.org/10.2174/156652312800099607 |
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
-
The Delivery of Biologically Active (Therapeutic) Peptides and Proteins into Cells
Current Medicinal Chemistry Cancer Gene Therapy by Adenovirus-Mediated Gene Transfer
Current Gene Therapy Hedgehog Signaling and Urological Cancers
Current Drug Targets Potentials and Challenges of Active Targeting at the Tumor Cells by Engineered Polymeric Nanoparticles
Current Pharmaceutical Biotechnology EGFR-Targeting Monoclonal Antibodies in Head and Neck Cancer
Current Cancer Drug Targets Saponins-uptake and Targeting Issues for Brain-specific Delivery for Enhanced Cell Death Induction in Glioblastoma
Letters in Drug Design & Discovery Pharmacophore Development and SAR Studies of Imidazoline Receptor Ligands
Mini-Reviews in Medicinal Chemistry The Functional Role of Long Non-coding RNA UCA1 in Human Multiple Cancers: a Review Study
Current Molecular Medicine Receptor Tyrosine Kinases Take a Direct Route to Mitochondria: An Overview
Current Protein & Peptide Science Modifications of Cell Signalling and Redox Balance by Targeting Protein Acetylation Using Natural and Engineered Molecules: Implications in Cancer Therapy
Current Topics in Medicinal Chemistry Integrins as Novel Drug Targets for Overcoming Innate Drug Resistance
Current Cancer Drug Targets Molecular Targeted Approaches to Cancer Therapy and Prevention Using Chalcones
Current Cancer Drug Targets Transplantation of Adipose Tissue-Derived Stem Cells for Treatment of Focal Cerebral Ischemia
Current Neurovascular Research Biological and Clinical Significance of Polymorphisms in NAD(P)H: Quinone Oxidoreductase 1 (NQO1)
Current Pharmacogenomics Versatility of Cancer Associated Fibroblasts: Commendable Targets for Anti-tumor Therapy
Current Drug Targets Design of Magnetic Nanoparticles-Assisted Drug Delivery System
Current Pharmaceutical Design Astrocytes: Implications for Neuroinflammatory Pathogenesis of Alzheimers Disease
Current Alzheimer Research Computational Evaluation and In Vitro Validation of New Epidermal Growth Factor Receptor Inhibitors
Current Topics in Medicinal Chemistry Targeting the Wingless Signaling Pathway with Natural Compounds as Chemopreventive or Chemotherapeutic Agents
Current Pharmaceutical Biotechnology Urokinase Receptor and Integrin Interactions
Current Pharmaceutical Design