Abstract
Ciliary neurotrophic factor (CNTF) is a cytokine with neurotrophic activity across a broad spectrum of peripheral and central nervous system (CNS) cells. While its therapeutic potential for CNS diseases has been clear for sometime, the blood brain barrier (BBB) hinders the systemic delivery of CNTF and direct bolus injections are not suitable due to the short half-life of CNTF. One means of overcoming the BBB while providing continuous delivery of CNTF is with immunoisolated cellular implants that produce and deliver CNTF directly to the region of interest. Cells can be protected from host rejection by encapsulating, or surrounding, them within an immunoisolatory, semipermeable membrane that admits oxygen and required nutrients and releases bioactive cell secretions, but restricts passage of larger cytotoxic agents from the host immune defense system. The selective membrane eliminates the need for chronic immunosuppression of the host and allows the implanted cells to be obtained from nonhuman sources. In this review we discuss cell immunoisolation for Huntingtons disease and retinitis pigmentosa. These two indications are highlighted because of extensive pre-clinical data supporting the general concept and recent clinical data that both strengthens the theoretical role of CNTF for treating neurodegeneration and justifies additional clinical evaluation in these and other diseases.
Keywords: cell survival, amyotrophic lateral sclerosis (ALS), GABAergic, neuroprotective, Baby hamster kidney fibroblast cells (BHK), Transplanted Cells
Current Gene Therapy
Title: Intracompartmental Delivery of CNTF as Therapy for Huntingtons Disease and Retinitis Pigmentosa
Volume: 6 Issue: 1
Author(s): Dwaine F. Emerich and Christopher G. Thanos
Affiliation:
Keywords: cell survival, amyotrophic lateral sclerosis (ALS), GABAergic, neuroprotective, Baby hamster kidney fibroblast cells (BHK), Transplanted Cells
Abstract: Ciliary neurotrophic factor (CNTF) is a cytokine with neurotrophic activity across a broad spectrum of peripheral and central nervous system (CNS) cells. While its therapeutic potential for CNS diseases has been clear for sometime, the blood brain barrier (BBB) hinders the systemic delivery of CNTF and direct bolus injections are not suitable due to the short half-life of CNTF. One means of overcoming the BBB while providing continuous delivery of CNTF is with immunoisolated cellular implants that produce and deliver CNTF directly to the region of interest. Cells can be protected from host rejection by encapsulating, or surrounding, them within an immunoisolatory, semipermeable membrane that admits oxygen and required nutrients and releases bioactive cell secretions, but restricts passage of larger cytotoxic agents from the host immune defense system. The selective membrane eliminates the need for chronic immunosuppression of the host and allows the implanted cells to be obtained from nonhuman sources. In this review we discuss cell immunoisolation for Huntingtons disease and retinitis pigmentosa. These two indications are highlighted because of extensive pre-clinical data supporting the general concept and recent clinical data that both strengthens the theoretical role of CNTF for treating neurodegeneration and justifies additional clinical evaluation in these and other diseases.
Export Options
About this article
Cite this article as:
Emerich F. Dwaine and Thanos G. Christopher, Intracompartmental Delivery of CNTF as Therapy for Huntingtons Disease and Retinitis Pigmentosa, Current Gene Therapy 2006; 6 (1) . https://dx.doi.org/10.2174/156652306775515547
DOI https://dx.doi.org/10.2174/156652306775515547 |
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
-
Kappa Receptor Bivalent Ligands
Current Topics in Medicinal Chemistry The Role of Autophagy in the Pathogenesis of Ischemic Stroke
Current Neuropharmacology Alpha-2 Adrenoceptor Ligands and Opioid Drugs: Pharmacological Interactions of Therapeutic Interest
Current Neuropharmacology Plasticity of T Cell Differentiation and Cytokine Signature: A Double-Edged Sword for Immune Responses
Immunology, Endocrine & Metabolic Agents in Medicinal Chemistry (Discontinued) Extrinsic Factors Promoting Insulin Producing Cell-Differentiation and Insulin Expression Enhancement-Hope for Diabetics.
Current Stem Cell Research & Therapy The Role of α7 Nicotinic Acetylcholine Receptors and α7-Specific Antibodies in Neuroinflammation Related to Alzheimer Disease
Current Pharmaceutical Design “Smart” Nanocarriers: A New Paradigm for Tumor Targeting Drug Delivery Systems
Drug Delivery Letters Oxidative stress and Parkinson’s disease: New hopes in treatment with herbal antioxidants
Current Pharmaceutical Design Proteasome Inhibitors: Recent Advances and New Perspectives In Medicinal Chemistry
Current Topics in Medicinal Chemistry Scope of Lipid Nanoparticles in Neuroscience: Impact on the Treatment of Neurodegenerative Diseases
Current Pharmaceutical Design Molecular Pharmacology of Malignant Pleural Mesothelioma: Challenges and Perspectives From Preclinical and Clinical Studies
Current Drug Targets Structure, Function, and Pathogenesis of SHP2 in Developmental Disorders and Tumorigenesis
Current Cancer Drug Targets Novel Multifunctional Anti-Alzheimer Drugs with Various CNS Neurotransmitter Targets and Neuroprotective Moieties
Current Alzheimer Research Cardiac Side Effects of Chemotherapy: State of Art and Strategies for a Correct Management
Current Vascular Pharmacology Development of PET Probes for Cancer Imaging
Current Topics in Medicinal Chemistry Biological Evaluation of 8-Hydroxyquinolines as Multi-Target Directed Ligands for Treating Alzheimer’s Disease
Current Alzheimer Research Peroxisome Proliferator Activated Receptor Gamma Coactivator 1 Alpha: An Emerging Target for Neuroprotection in Parkinson’s Disease
CNS & Neurological Disorders - Drug Targets Cancer-Homing Toxins
Current Pharmaceutical Design Fibroblast Growth Factors, Fibroblast Growth Factor Receptors, Diseases, and Drugs
Recent Patents on Cardiovascular Drug Discovery Inhibition of Monoamine Neurotransmitter Transporters andCentral Nervous System Stimulation Induced by Synthetic Local Anesthetics and Cocaine: A Comparative Review
Current Medicinal Chemistry - Central Nervous System Agents