AAV Targeting of Glial Cell Types in the Central and Peripheral Nervous System and Relevance to Human Gene Therapy

Different glial cell types are found throughout the central (CNS) and peripheral nervous system (PNS), where they have important functions. These cell types are also involved in nervous system pathology, playing roles in neurodegenerative disease and following trauma in the brain and spinal cord (as...

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Main Authors:	Simon J. O’Carroll, William H. Cook, Deborah Young
Format:	Article
Language:	English
Published:	Frontiers Media S.A. 2021-01-01
Series:	Frontiers in Molecular Neuroscience
Subjects:	gene therapy AAV glia astrocyte oligodendrocyte microglia
Online Access:	https://www.frontiersin.org/articles/10.3389/fnmol.2020.618020/full

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spelling	doaj-efc5e8ce04354d13aaf39d5472c1d6432021-01-11T05:05:38ZengFrontiers Media S.A.Frontiers in Molecular Neuroscience1662-50992021-01-011310.3389/fnmol.2020.618020618020AAV Targeting of Glial Cell Types in the Central and Peripheral Nervous System and Relevance to Human Gene TherapySimon J. O’Carroll0William H. Cook1Deborah Young2Spinal Cord Injury Research Group, Department of Anatomy and Medical Imaging, School of Medical Sciences, University of Auckland, Auckland, New ZealandMolecular Neurotherapeutics Group, Department of Pharmacology and Clinical Pharmacology, School of Medical Sciences, University of Auckland, Auckland, New ZealandMolecular Neurotherapeutics Group, Department of Pharmacology and Clinical Pharmacology, School of Medical Sciences, University of Auckland, Auckland, New ZealandDifferent glial cell types are found throughout the central (CNS) and peripheral nervous system (PNS), where they have important functions. These cell types are also involved in nervous system pathology, playing roles in neurodegenerative disease and following trauma in the brain and spinal cord (astrocytes, microglia, oligodendrocytes), nerve degeneration and development of pain in peripheral nerves (Schwann cells, satellite cells), retinal diseases (Müller glia) and gut dysbiosis (enteric glia). These cell type have all been proposed as potential targets for treating these conditions. One approach to target these cell types is the use of gene therapy to modify gene expression. Adeno-associated virus (AAV) vectors have been shown to be safe and effective in targeting cells in the nervous system and have been used in a number of clinical trials. To date, a number of studies have tested the use of different AAV serotypes and cell-specific promoters to increase glial cell tropism and expression. However, true glial-cell specific targeting for a particular glial cell type remains elusive. This review provides an overview of research into developing glial specific gene therapy and discusses some of the issues that still need to be addressed to make glial cell gene therapy a clinical reality.https://www.frontiersin.org/articles/10.3389/fnmol.2020.618020/fullgene therapyAAVgliaastrocyteoligodendrocytemicroglia
collection	DOAJ
language	English
format	Article
sources	DOAJ
author	Simon J. O’Carroll William H. Cook Deborah Young
spellingShingle	Simon J. O’Carroll William H. Cook Deborah Young AAV Targeting of Glial Cell Types in the Central and Peripheral Nervous System and Relevance to Human Gene Therapy Frontiers in Molecular Neuroscience gene therapy AAV glia astrocyte oligodendrocyte microglia
author_facet	Simon J. O’Carroll William H. Cook Deborah Young
author_sort	Simon J. O’Carroll
title	AAV Targeting of Glial Cell Types in the Central and Peripheral Nervous System and Relevance to Human Gene Therapy
title_short	AAV Targeting of Glial Cell Types in the Central and Peripheral Nervous System and Relevance to Human Gene Therapy
title_full	AAV Targeting of Glial Cell Types in the Central and Peripheral Nervous System and Relevance to Human Gene Therapy
title_fullStr	AAV Targeting of Glial Cell Types in the Central and Peripheral Nervous System and Relevance to Human Gene Therapy
title_full_unstemmed	AAV Targeting of Glial Cell Types in the Central and Peripheral Nervous System and Relevance to Human Gene Therapy
title_sort	aav targeting of glial cell types in the central and peripheral nervous system and relevance to human gene therapy
publisher	Frontiers Media S.A.
series	Frontiers in Molecular Neuroscience
issn	1662-5099
publishDate	2021-01-01
description	Different glial cell types are found throughout the central (CNS) and peripheral nervous system (PNS), where they have important functions. These cell types are also involved in nervous system pathology, playing roles in neurodegenerative disease and following trauma in the brain and spinal cord (astrocytes, microglia, oligodendrocytes), nerve degeneration and development of pain in peripheral nerves (Schwann cells, satellite cells), retinal diseases (Müller glia) and gut dysbiosis (enteric glia). These cell type have all been proposed as potential targets for treating these conditions. One approach to target these cell types is the use of gene therapy to modify gene expression. Adeno-associated virus (AAV) vectors have been shown to be safe and effective in targeting cells in the nervous system and have been used in a number of clinical trials. To date, a number of studies have tested the use of different AAV serotypes and cell-specific promoters to increase glial cell tropism and expression. However, true glial-cell specific targeting for a particular glial cell type remains elusive. This review provides an overview of research into developing glial specific gene therapy and discusses some of the issues that still need to be addressed to make glial cell gene therapy a clinical reality.
topic	gene therapy AAV glia astrocyte oligodendrocyte microglia
url	https://www.frontiersin.org/articles/10.3389/fnmol.2020.618020/full
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AAV Targeting of Glial Cell Types in the Central and Peripheral Nervous System and Relevance to Human Gene Therapy

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