Abstract
Gene therapy has been a clinical possibility since 1989 and the steadily increasing number of clinical trials now includes strategies targeting neurodegenerative conditions such as lysosomal storage disease, multiple sclerosis, Alzheimer s and, Parkinsons disease. In spite of lack of knowledge of the molecular causes of these diseases, results so far in these trials have been promising. Thus there is gaining confidence in the potential to develop effective treatments based on gene transfer for neurological diseases in the near future. Furthermore, the accelerating progress in knowledge of the molecular pathologies of neurogenetic disorders, including rare diseases such as the ataxias, makes them even more amenable to gene therapy. Here we review recent preclinical studies relevant to gene therapy of ataxias and discuss developments needed to bring these strategies into the clinic.
Keywords: Viral vectors, gene transfer, gene delivery, herpesvirus, Friedreich's ataxia, neuromuscular disorders, neurodegeneratio
Current Gene Therapy
Title: Gene Therapy Approaches to Ataxias
Volume: 9 Issue: 1
Author(s): Filip Lim and Javier Diaz-Nido
Affiliation:
Keywords: Viral vectors, gene transfer, gene delivery, herpesvirus, Friedreich's ataxia, neuromuscular disorders, neurodegeneratio
Abstract: Gene therapy has been a clinical possibility since 1989 and the steadily increasing number of clinical trials now includes strategies targeting neurodegenerative conditions such as lysosomal storage disease, multiple sclerosis, Alzheimer s and, Parkinsons disease. In spite of lack of knowledge of the molecular causes of these diseases, results so far in these trials have been promising. Thus there is gaining confidence in the potential to develop effective treatments based on gene transfer for neurological diseases in the near future. Furthermore, the accelerating progress in knowledge of the molecular pathologies of neurogenetic disorders, including rare diseases such as the ataxias, makes them even more amenable to gene therapy. Here we review recent preclinical studies relevant to gene therapy of ataxias and discuss developments needed to bring these strategies into the clinic.
Export Options
About this article
Cite this article as:
Lim Filip and Diaz-Nido Javier, Gene Therapy Approaches to Ataxias, Current Gene Therapy 2009; 9 (1) . https://dx.doi.org/10.2174/156652309787354649
DOI https://dx.doi.org/10.2174/156652309787354649 |
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
-
Patent Selections:
Recent Patents on Endocrine, Metabolic & Immune Drug Discovery Leber's Hereditary Optic Neuropathy: Novel Views and Persisting Challenges
CNS & Neurological Disorders - Drug Targets Postponing Motherhood: A Demographic and Contemporary Issue
Current Women`s Health Reviews The Progress Towards the Development of DHQO Derivatives and Related Analogues with Inotropic Effects
Mini-Reviews in Medicinal Chemistry Assessment of Cardiac Sympathetic Innervation in Heart Failure and Lethal Arrhythmias: Therapeutic and Prognostic Implications
Current Cardiology Reviews Oxidative Stress in Diabetic Nephropathy
Current Medicinal Chemistry The Impact of Small Heat Shock Proteins (HspBs) in Alzheimer’s and Other Neurological Diseases
Current Pharmaceutical Design Effects of Metabolic Approach in Diabetic Patients with Coronary Artery Disease
Current Pharmaceutical Design Combating Neurodegenerative Diseases with the Plant Alkaloid Berberine: Molecular Mechanisms and Therapeutic Potential
Current Neuropharmacology Editorial [Hot Topic : Current Practice and trends in echocardiography in Critical Care, Anesthesiology and Emergency Medicine (Guest Editor: Paulo Marcelino)]
Current Cardiology Reviews A Review on Electrocardiographic Changes in Diabetic Patients
Current Diabetes Reviews EDITORIAL [Hot Topic: Diabetes Cardiovascular Complications (Guest Editor: Costanza Emanueli)]
Endocrine, Metabolic & Immune Disorders - Drug Targets One Special Question to Start with: Can HIF/NFkB be a Target in Inflammation?
Endocrine, Metabolic & Immune Disorders - Drug Targets Melatonin Alleviates Pyroptosis of Retinal Neurons Following Acute Intraocular Hypertension
CNS & Neurological Disorders - Drug Targets The Biological and Clinical Activity of Anti-Malarial Drugs In Autoimmune Disorders
Current Rheumatology Reviews microRNA as Biomarkers and Regulator of Cardiovascular Development and Disease
Current Pharmaceutical Design Link between Metabolic Syndrome and Insulin Resistance
Current Vascular Pharmacology Diabetic Cardiomyopathy: Electromechanical Cellular Alterations
Current Vascular Pharmacology A Review of Therapeutic Effects of Curcumin
Current Pharmaceutical Design Tandem Mass Spectrometry Newborn Screening for Inborn Errors of Intermediary Metabolism: Abnormal Profile Interpretation
Current Medicinal Chemistry