Abstract
Exosomes are a subtype of membrane vesicle released from the endocytic compartment of live cells. They play an important role in endogenous cell-to-cell communication. Previously shown to be capable of traversing biological barriers and to naturally transport functional nucleic acids between cells, they potentially represent a novel and exciting drug delivery vehicle for the field of gene therapy. Existing delivery vehicles are limited by concerns regarding their safety, toxicity and efficacy. In contrast, exosomes, as a natural cell-derived nanocarrier, are immunologically inert if purified from a compatible cell source and possess an intrinsic ability to cross biological barriers. Already utilised in a number of clinical trials, exosomes appear to be well-tolerated, even following repeat administration. Recent studies have shown that exosomes may be used to encapsulate and protect exogenous oligonucleotides for delivery to target cells. They therefore may be valuable for the delivery of RNA interference and microRNA regulatory molecules in addition to other singlestranded oligonucleotides. Prior to clinical translation, this nanotechnology requires further development by refinement of isolation, purification, loading, delivery and targeting protocols. Thus, exosome-mediated nanodelivery is highly promising and may fill the void left by current delivery methods for systemic gene therapy.
Keywords: Exosome, gene delivery, gene therapy, nanomedicine, RNAi, stem cells, vector, nanotechnology.
Current Gene Therapy
Title:Exosomes and the Emerging Field of Exosome-Based Gene Therapy
Volume: 12 Issue: 4
Author(s): Aisling J. O’Loughlin, Caroline A. Woffindale and Matthew J.A. Wood
Affiliation:
Keywords: Exosome, gene delivery, gene therapy, nanomedicine, RNAi, stem cells, vector, nanotechnology.
Abstract: Exosomes are a subtype of membrane vesicle released from the endocytic compartment of live cells. They play an important role in endogenous cell-to-cell communication. Previously shown to be capable of traversing biological barriers and to naturally transport functional nucleic acids between cells, they potentially represent a novel and exciting drug delivery vehicle for the field of gene therapy. Existing delivery vehicles are limited by concerns regarding their safety, toxicity and efficacy. In contrast, exosomes, as a natural cell-derived nanocarrier, are immunologically inert if purified from a compatible cell source and possess an intrinsic ability to cross biological barriers. Already utilised in a number of clinical trials, exosomes appear to be well-tolerated, even following repeat administration. Recent studies have shown that exosomes may be used to encapsulate and protect exogenous oligonucleotides for delivery to target cells. They therefore may be valuable for the delivery of RNA interference and microRNA regulatory molecules in addition to other singlestranded oligonucleotides. Prior to clinical translation, this nanotechnology requires further development by refinement of isolation, purification, loading, delivery and targeting protocols. Thus, exosome-mediated nanodelivery is highly promising and may fill the void left by current delivery methods for systemic gene therapy.
Export Options
About this article
Cite this article as:
O’Loughlin J. Aisling, Woffindale A. Caroline and Wood J.A. Matthew, Exosomes and the Emerging Field of Exosome-Based Gene Therapy, Current Gene Therapy 2012; 12 (4) . https://dx.doi.org/10.2174/156652312802083594
DOI https://dx.doi.org/10.2174/156652312802083594 |
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
-
Relationship Between the Pharmacokinetics of Levosimendan and Its Effects on Cardiovascular System
Current Drug Metabolism Therapeutic Applications of Crocus sativus L. (Saffron): A Review
The Natural Products Journal Adenosine Neuromodulation and Traumatic Brain Injury
Current Neuropharmacology Recent Patents, Regulatory Issues, and Toxicity of Nanoparticles in Neuronal Disorders
Current Drug Metabolism Female Infertility and Antioxidants
Current Women`s Health Reviews Adipose Tissue Derived Stem Cells Secretome: Soluble Factors and Their Roles in Regenerative Medicine
Current Stem Cell Research & Therapy Beneficial Role of Vitexin and Isovitexin Flavonoids in the Vascular Endothelium and Cardiovascular System
Current Nutraceuticals Cardiovascular Effects of Modulators of Soluble Guanylyl Cyclase Activity
Cardiovascular & Hematological Agents in Medicinal Chemistry From Polypharmacology to Target Specificity: The Case of PARP Inhibitors
Current Topics in Medicinal Chemistry The Neuroprotective Mechanism of Erythropoietin-TAT Fusion Protein Against Neurodegeneration from Ischemic Brain Injury
CNS & Neurological Disorders - Drug Targets Furocoumarins from Cnidium monnieri Act as Peroxisome Proliferatoractivated R and Farnesoid X Receptor Agonists
Mini-Reviews in Organic Chemistry Why Multiples of 21? Why does Selenoprotein P Contain Multiple Selenocysteine Residues?
Current Nutraceuticals Stem Cell Gene Therapy for Chronic Renal Failure
Current Gene Therapy Peroxisome Proliferator-Activated Receptors: The Nutritionally Controlled Molecular Networks that Integrate Inflammation, Immunity and Metabolism
Current Nutrition & Food Science Recent Advances in Optimal Adjunctive Antithrombotic Therapy in STEMI Patients Undergoing Primary Angioplasty: An Overview
Current Vascular Pharmacology Carvacrol as a Potential Neuroprotective Agent for Neurological Diseases: A Systematic Review Article
CNS & Neurological Disorders - Drug Targets Medication-Induced Nephrotoxicity in Older Patients
Current Drug Metabolism Endothelial Dysfunction: Methods of Assessment and Application to Hypertension
Current Pharmaceutical Design Reduction of Oxidative/Nitrosative Stress in Brain and its Involvement in the Neuroprotective Effect of n-3 PUFA in Alzheimer’s Disease
Current Alzheimer Research Complement and Complement Regulatory Proteins as Potential Molecular Targets for Vascular Diseases
Current Pharmaceutical Design