Abstract
Therapy for Parkinsons disease (PD), a common neurological disorder characterized by pathological degeneration of the nigrostriatal dopaminergic system, remains unsatisfactory. Gene therapy is considered one of the most promising approaches to developing a novel effective treatment for PD. Among the numerous candidate genes that have been tested as therapeutic agents, those encoding tyrosine hydroxylase, guanosine triphosphate cyclohydrolase I and aromatic L-amino acid decarboxylase all boost dopamine production, while glial cell line-derived neurotrophic factor promotes the survival of dopaminergic neurons and is generally believed to possess the greatest potential for successful restoration of the dopaminergic system. The genes encoding vesicular monoamine transporter-2 and glutamic acid decarboxylase have also produced therapeutic effects in animal models of PD. Both viral and non-viral vectors, each with its particular advantages and disadvantages, have been used to deliver these genes into the brain. Whether or not regulatable expression systems are essential to successful gene therapy for PD remains a critical issue in the clinical application of this emerging treatment. Here we review the current status of gene therapy for PD, including the application of control systems for transgene expression in the brain.
Keywords: parkinsons disease, gene therapy, glial cell line-derived neurotrophic factor, viral vectors, tyrosine hydroxylase, guanosine triphosphate cyclohydrolase I
Current Gene Therapy
Title: Gene Therapy for Parkinsons Disease: Progress and Challenges
Volume: 5 Issue: 1
Author(s): Qin Chen, Yi He and Keyi Yang
Affiliation:
Keywords: parkinsons disease, gene therapy, glial cell line-derived neurotrophic factor, viral vectors, tyrosine hydroxylase, guanosine triphosphate cyclohydrolase I
Abstract: Therapy for Parkinsons disease (PD), a common neurological disorder characterized by pathological degeneration of the nigrostriatal dopaminergic system, remains unsatisfactory. Gene therapy is considered one of the most promising approaches to developing a novel effective treatment for PD. Among the numerous candidate genes that have been tested as therapeutic agents, those encoding tyrosine hydroxylase, guanosine triphosphate cyclohydrolase I and aromatic L-amino acid decarboxylase all boost dopamine production, while glial cell line-derived neurotrophic factor promotes the survival of dopaminergic neurons and is generally believed to possess the greatest potential for successful restoration of the dopaminergic system. The genes encoding vesicular monoamine transporter-2 and glutamic acid decarboxylase have also produced therapeutic effects in animal models of PD. Both viral and non-viral vectors, each with its particular advantages and disadvantages, have been used to deliver these genes into the brain. Whether or not regulatable expression systems are essential to successful gene therapy for PD remains a critical issue in the clinical application of this emerging treatment. Here we review the current status of gene therapy for PD, including the application of control systems for transgene expression in the brain.
Export Options
About this article
Cite this article as:
Chen Qin, He Yi and Yang Keyi, Gene Therapy for Parkinsons Disease: Progress and Challenges, Current Gene Therapy 2005; 5 (1) . https://dx.doi.org/10.2174/1566523052997505
DOI https://dx.doi.org/10.2174/1566523052997505 |
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
-
Screening Neuroprotective Agents Through 4-hydroxynonenal, Ethanol, High Glucose, Homocysteine, Okadaic Acid, Rotenone, and Oxygen-Glucose Deprivation Induced PC12 Injury Models: A Review
Current Psychopharmacology TP73, An Under-Appreciated Player in Non-Hodgkin Lymphoma Pathogenesis and Management
Current Molecular Medicine Therapeutic Potential of Inhibiting Brutons Tyrosine Kinase, (BTK)
Current Pharmaceutical Design Do mtDNA Mutations Participate in the Pathogenesis of Sporadic Parkinsons Disease?
Current Genomics EDITORIAL [Hot topic: New Therapeutic Advances and Perspectives in Tumour Angiogenesis (Guest Editor: Eddy Pasquier)]
Current Cancer Drug Targets Pathogenic Mechanisms and Therapeutic Strategies in Spinobulbar Muscular Atrophy
CNS & Neurological Disorders - Drug Targets Emerging RNA-based Drugs: siRNAs, microRNAs and Derivates
Central Nervous System Agents in Medicinal Chemistry Polysialyltransferase: A New Target in Metastatic Cancer
Current Cancer Drug Targets The Use of Cytokines and Chemokines in the Cancer Immunotherapy
Recent Patents on Anti-Cancer Drug Discovery Molecular Mechanisms, Biological Actions, and Neuropharmacology of the Growth-Associated Protein GAP-43
Current Neuropharmacology Oxazole-Based Compounds As Anticancer Agents
Current Medicinal Chemistry Role of Flavonoids in Future Anticancer Therapy by Eliminating the Cancer Stem Cells
Current Stem Cell Research & Therapy Mitocans: Mitochondrial Targeted Anti-Cancer Drugs as Improved Therapies and Related Patent Documents
Recent Patents on Anti-Cancer Drug Discovery Calcium Ion – The Key Player in Cerebral Ischemia
Current Medicinal Chemistry Drug Therapy of Neuropathic Pain: Current Developments and Future Perspectives
Current Drug Targets High-Throughput Screening Technologies for Botulinum Neurotoxins
Current Topics in Medicinal Chemistry Effect of Phosphorylation and Aggregation on Tau Binding to Dna
Protein & Peptide Letters Gene Expression Signatures of Lymph Node Metastasis in Oral Cancer: Molecular Characteristics and Clinical Significances
Current Cancer Therapy Reviews Genetic Editing and Pharmacogenetics in Current And Future Therapy Of Neurocognitive Disorders
Current Alzheimer Research Neurotoxins: Free Radical Mechanisms and Melatonin Protection
Current Neuropharmacology