Abstract
Therapeutic delivery to the central nervous system has challenged scientists and clinicians due to the difficulty in delivering molecules and genes in an efficient manner across the blood brain barrier (BBB). This has particularly hampered efforts to deliver therapeutics to widely dispersed neurons that perish in diseases such as Amyotrophic Lateral Sclerosis (ALS), a disease affecting motor neurons throughout the brainstem and the entire spinal cord. Gene therapy has offered several potential routes to overcome the difficulties in delivering therapeutics to the brain and spinal cord. Adenoassociated viral vectors (AAV) have taken center stage for gene delivery to the central nervous system, given their ability to express genes in post mitotic cells for long periods with minimal to no toxicity. This review will focus on recent approaches to treat motor neuron disease, in particular ALS using AAV vectors.
Keywords: Gene therapy, motor neuron disease, Amyotrophic Lateral Sclerosis (ALS), Adeno-associated virus (AAV), blood brain barrier (BBB)
Current Gene Therapy
Title: AAV as a Gene Transfer Vector for the Treatment of Neurological Disorders: Novel Treatment Thoughts for ALS
Volume: 9 Issue: 5
Author(s): Mark E. Hester, Kevin D. Foust, Rita W. Kaspar and Brian K. Kaspar
Affiliation:
Keywords: Gene therapy, motor neuron disease, Amyotrophic Lateral Sclerosis (ALS), Adeno-associated virus (AAV), blood brain barrier (BBB)
Abstract: Therapeutic delivery to the central nervous system has challenged scientists and clinicians due to the difficulty in delivering molecules and genes in an efficient manner across the blood brain barrier (BBB). This has particularly hampered efforts to deliver therapeutics to widely dispersed neurons that perish in diseases such as Amyotrophic Lateral Sclerosis (ALS), a disease affecting motor neurons throughout the brainstem and the entire spinal cord. Gene therapy has offered several potential routes to overcome the difficulties in delivering therapeutics to the brain and spinal cord. Adenoassociated viral vectors (AAV) have taken center stage for gene delivery to the central nervous system, given their ability to express genes in post mitotic cells for long periods with minimal to no toxicity. This review will focus on recent approaches to treat motor neuron disease, in particular ALS using AAV vectors.
Export Options
About this article
Cite this article as:
Hester E. Mark, Foust D. Kevin, Kaspar W. Rita and Kaspar K. Brian, AAV as a Gene Transfer Vector for the Treatment of Neurological Disorders: Novel Treatment Thoughts for ALS, Current Gene Therapy 2009; 9 (5) . https://dx.doi.org/10.2174/156652309789753383
DOI https://dx.doi.org/10.2174/156652309789753383 |
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
-
Insight γ-Secretase: Structure, Function, and Role in Alzheimer’s Disease
Current Drug Targets Cdk5 in the Adult Non-Demented Brain
Current Drug Targets - CNS & Neurological Disorders Shikimate Kinase (EC 2.7.1.71) from Mycobacterium tuberculosis: Kinetics and Structural Dynamics of a Potential Molecular Target for Drug Development
Current Medicinal Chemistry The Potential Role of Sirtuins Regarding the Effects of Exercise on Aging- Related Diseases
Current Aging Science Does a Pro-Inflammatory Process Precede Alzheimers Disease and Mild Cognitive Impairment?
Current Alzheimer Research Quantitative Ultrasound of Orofacial Muscles in Infants from 6 Months to 5 Years: Collecting Normal Values
Current Medical Imaging Evolving Therapeutic Targets in Ischemic Stroke: A Concise Review
Current Drug Targets Magnetic Materials for the Selective Analysis of Peptide and Protein Biomarkers
Current Medicinal Chemistry Pharmaceutical Targeting of the Brain
Current Pharmaceutical Design 2,5-Diketopiperazines as Neuroprotective Agents
Mini-Reviews in Medicinal Chemistry The Impact of Small Heat Shock Proteins (HspBs) in Alzheimer’s and Other Neurological Diseases
Current Pharmaceutical Design Stem Cell Regenerative Potential Combined with Nanotechnology and Tissue Engineering for Myocardial Regeneration
Current Stem Cell Research & Therapy The Implications of Sortilin/Vps10p Domain Receptors in Neurological and Human Diseases
CNS & Neurological Disorders - Drug Targets Current Options in the Treatment of Mitochondrial Diseases
Recent Patents on CNS Drug Discovery (Discontinued) Therapeutic Potential of Janus Kinase 3 (JAK3) Inhibitors
Current Pharmaceutical Design Role of Kynurenines in the Central and Peripherial Nervous Systems
Current Neurovascular Research D-amino Acids as Novel Blood-based Biomarkers
Current Medicinal Chemistry Stem Cells with Neurogenic Potential and Steroid Hormones
Current Topics in Medicinal Chemistry Aquaporins and Neurodegenerative Diseases
Current Neuropharmacology Carotenoids of Microalgae Used in Food Industry and Medicine
Mini-Reviews in Medicinal Chemistry