Abstract
Targeted gene therapy aims at achieving the expression of therapeutic transgenes in specific and restricted cell populations, thus sparing all other cells of the unwanted effects of the gene product. This strategy is particularly appealing for therapy of the central nervous system (CNS), where many different cell types exist, and where the inappropriate expression of a molecule can produce grave consequences. To accomplish the objectives of targeted gene therapy, two different approaches have been developed. The first one consists in creating vectors that will deliver the transgene exclusively to the selected cells, that is manipulating the transductional capacities of the vector, and the second one is based on the transcriptional properties of the transgene, so that it will only be expressed in cells where the appropriate transcriptional machinery is present. Reaching the goals of targeted gene expression will greatly increase the specificity and safety of gene therapy, thus getting us closer to the fulfillment of the expectations generated by this new branch of molecular medicine.
Keywords: promoter, viral vector, adenovirus, gene transfer, retrovirus, nervous system, transduction, gene transcription, gene therapy
Current Gene Therapy
Title: Gene Therapy Targeting in the Central Nervous System
Volume: 3 Issue: 2
Author(s): Jorge A. Benitez and Jose Segovia
Affiliation:
Keywords: promoter, viral vector, adenovirus, gene transfer, retrovirus, nervous system, transduction, gene transcription, gene therapy
Abstract: Targeted gene therapy aims at achieving the expression of therapeutic transgenes in specific and restricted cell populations, thus sparing all other cells of the unwanted effects of the gene product. This strategy is particularly appealing for therapy of the central nervous system (CNS), where many different cell types exist, and where the inappropriate expression of a molecule can produce grave consequences. To accomplish the objectives of targeted gene therapy, two different approaches have been developed. The first one consists in creating vectors that will deliver the transgene exclusively to the selected cells, that is manipulating the transductional capacities of the vector, and the second one is based on the transcriptional properties of the transgene, so that it will only be expressed in cells where the appropriate transcriptional machinery is present. Reaching the goals of targeted gene expression will greatly increase the specificity and safety of gene therapy, thus getting us closer to the fulfillment of the expectations generated by this new branch of molecular medicine.
Export Options
About this article
Cite this article as:
Benitez A. Jorge and Segovia Jose, Gene Therapy Targeting in the Central Nervous System, Current Gene Therapy 2003; 3 (2) . https://dx.doi.org/10.2174/1566523034578429
DOI https://dx.doi.org/10.2174/1566523034578429 |
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
-
Nanotechnology and Brain Tumors Drug Delivery
Recent Patents on Nanomedicine Biomarkers of Angiogenesis and their Role in Patient Selection for Antiangiogenic Therapy
Current Angiogenesis (Discontinued) Hedgehog Target Genes: Mechanisms of Carcinogenesis Induced by Aberrant Hedgehog Signaling Activation
Current Molecular Medicine Review on Documented Medicinal Plants used for the Treatment of Cancer
Current Traditional Medicine The Use of Immortalized Cell Lines in GPCR Screening: The Good, Bad and Ugly.
Combinatorial Chemistry & High Throughput Screening Antitumor Effect of Butanoylated Heparin with Low Anticoagulant Activity on Lung Cancer Growth in Mice and Rats
Current Cancer Drug Targets The Molecular Targets of Cannabinoids in the Treatment of Cancer and Inflammation
Current Pharmaceutical Design Current Inhibitors of Checkpoint Kinase 2
Current Medicinal Chemistry Triazene Compounds in the Treatment of Acute Myeloid Leukemia: A Short Review and a Case Report
Current Medicinal Chemistry Anti-Cancer Potential of Some Commonly Used Drugs
Current Pharmaceutical Design Pharmacological Tools to Activate Microglia and their Possible use to Study Neural Network Patho-physiology
Current Neuropharmacology Pharmacogenetics of Non-small Cell Lung Cancer (NSCLC): Time to "Work it Out"?
Current Pharmaceutical Design Recent Developments of 18F-FET PET in Neuro-oncology
Current Medicinal Chemistry MicroRNAs in Neuroblastoma: Biomarkers with Therapeutic Potential
Current Medicinal Chemistry Osteopontin as a Potential Therapeutic Target for Ischemic Stroke
Current Drug Delivery Suppressing Glioblastoma Stem Cell Function by Aldehyde Dehydrogenase Inhibition with Chloramphenicol or Disulfiram as a New Treatment Adjunct: A Hypothesis
Current Stem Cell Research & Therapy Targeting Protein Tyrosine Phosphatases for Anticancer Drug Discovery
Current Pharmaceutical Design HtrA Serine Proteases as Potential Therapeutic Targets in Cancer
Current Cancer Drug Targets Human Growth Hormone Induced Cholestatic Hepatitis in a Growth Hormone Deficient Patient with Short Stature
Current Drug Safety Inhibition of Tumor Angiogenesis by Antibodies, Synthetic Small Molecules and Natural Products
Current Medicinal Chemistry