Abstract
Efficient therapeutic protein delivery is a challenging task in several disease contexts and particularly when the CNS is concerned. Different approaches for brain-directed delivery have been thus far investigated, including direct injection of molecules or of their coding information carried by dedicated vector systems within the brain parenchyma or in the ventricular space, intravenous systemic administration of molecules/vectors modified to target and cross the blood-brainbarrier, and exploitation of allogeneic and/or autologous and genetically modified cells as vehicles for the therapeutic of interest. Among these, we here review one of the most promising approaches based on hematopoietic stem cells, taking advantage of lysosomal storage disorders as representative disease setting.
Keywords: Hematopoietic stem cells, gene therapy, central nervous system, lysosomal storage disorders, blood-brain-barrier, protein delivery, brain parenchyma, ventricular space
Current Gene Therapy
Title:Genetically-Modified Hematopoietic Stem Cells and their Progeny for Widespread and Efficient Protein Delivery to Diseased Sites: The Case of Lysosomal Storage Disorders
Volume: 12 Issue: 5
Author(s): Alessandra Biffi
Affiliation:
Keywords: Hematopoietic stem cells, gene therapy, central nervous system, lysosomal storage disorders, blood-brain-barrier, protein delivery, brain parenchyma, ventricular space
Abstract: Efficient therapeutic protein delivery is a challenging task in several disease contexts and particularly when the CNS is concerned. Different approaches for brain-directed delivery have been thus far investigated, including direct injection of molecules or of their coding information carried by dedicated vector systems within the brain parenchyma or in the ventricular space, intravenous systemic administration of molecules/vectors modified to target and cross the blood-brainbarrier, and exploitation of allogeneic and/or autologous and genetically modified cells as vehicles for the therapeutic of interest. Among these, we here review one of the most promising approaches based on hematopoietic stem cells, taking advantage of lysosomal storage disorders as representative disease setting.
Export Options
About this article
Cite this article as:
Biffi Alessandra, Genetically-Modified Hematopoietic Stem Cells and their Progeny for Widespread and Efficient Protein Delivery to Diseased Sites: The Case of Lysosomal Storage Disorders, Current Gene Therapy 2012; 12 (5) . https://dx.doi.org/10.2174/156652312802762572
DOI https://dx.doi.org/10.2174/156652312802762572 |
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
-
Immunotherapy for Conformational Diseases
Current Pharmaceutical Design [General Articles] Cannabinoids: Mechanisms and Therapeutic Applications in the CNS
Current Medicinal Chemistry Osteopontin; as a Target Molecule for the Treatment of Inflammatory Diseases
Current Drug Targets GEMSP: A New Therapeutic Approach to Multiple Sclerosis
Central Nervous System Agents in Medicinal Chemistry Role of Interleukin-21 in Inflammation and Allergy
Inflammation & Allergy - Drug Targets (Discontinued) Recent Advances on the Roles of NO in Cancer and Chronic Inflammatory Disorders
Current Medicinal Chemistry Editorial (Thematic Issue: GABAergic Modulation as Treatment Strategy: Consideration of Several Diseases)
Current Pharmaceutical Design IL-10 Producing Regulatory B Cells in Mice and Humans: State of the Art
Current Molecular Medicine Curcuminoids in Neurodegenerative Diseases
Recent Patents on CNS Drug Discovery (Discontinued) Lipoic Acid: A Novel Therapeutic Approach for Multiple Sclerosis and Other Chronic Inflammatory Diseases of the CNS
Endocrine, Metabolic & Immune Disorders - Drug Targets Antiinflammatory Activities of CGRP Modulating Innate Immune Responses in Health and Disease
Current Protein & Peptide Science Polyphenols as Potential Therapeutics for Pain and Inflammation in Spinal Cord Injury
Current Molecular Pharmacology Neurotuberculosis: An Overview
Central Nervous System Agents in Medicinal Chemistry Glucocorticoid Analogues: Potential Therapeutic Alternatives for Treating Inflammatory Muscle Diseases
Endocrine, Metabolic & Immune Disorders - Drug Targets Allergen-Induced Inflammation
Anti-Inflammatory & Anti-Allergy Agents in Medicinal Chemistry Discovery of Inhibitors of Protein-Protein Interactions from Combinatorial Libraries
Current Topics in Medicinal Chemistry Promises of Nanotechnology for Drug Delivery to Brain in Neurodegenerative Diseases
Current Nanoscience Failure of Immune Homeostasis - The Consequences of Under and Over Reactivity
Current Drug Targets - Immune, Endocrine & Metabolic Disorders TLR7/9 Antagonists as Therapeutics for Immune-Mediated Inflammatory Disorders
Inflammation & Allergy - Drug Targets (Discontinued) Mesenchymal Stem Cells Neuronal Differentiation Ability: A Real Perspective for Nervous System Repair?
Current Stem Cell Research & Therapy