Specific gene expression in unmyelinated dorsal root ganglion neurons in nonhuman primates by intra-nerve injection of AAV 6 vector

Adeno-associated virus 6 (AAV6) has been proposed as a potential vector candidate for specific gene expression in pain-related dorsal root ganglion (DRG) neurons, but this has not been confirmed in nonhuman primates. The aim of our study was to analyze the transduction efficiency and target specific...

Full description

Bibliographic Details
Main Authors: Moeko Kudo, Sidikejiang Wupuer, Maki Fujiwara, Yuko Saito, Shinji Kubota, Ken-ichi Inoue, Masahiko Takada, Kazuhiko Seki
Format: Article
Language:English
Published: Elsevier 2021-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121001261
_version_ 1828956482267250688
author Moeko Kudo
Sidikejiang Wupuer
Maki Fujiwara
Yuko Saito
Shinji Kubota
Ken-ichi Inoue
Masahiko Takada
Kazuhiko Seki
author_facet Moeko Kudo
Sidikejiang Wupuer
Maki Fujiwara
Yuko Saito
Shinji Kubota
Ken-ichi Inoue
Masahiko Takada
Kazuhiko Seki
author_sort Moeko Kudo
collection DOAJ
description Adeno-associated virus 6 (AAV6) has been proposed as a potential vector candidate for specific gene expression in pain-related dorsal root ganglion (DRG) neurons, but this has not been confirmed in nonhuman primates. The aim of our study was to analyze the transduction efficiency and target specificity of this viral vector in the common marmoset by comparing it with those in the rat. When green fluorescent protein-expressing serotype-6 vector was injected into the sciatic nerve, the efficiency of gene expression in DRG neurons was comparable in both species. We found that the serotype-6 vector was largely specific to the pain-related ganglion neurons in the marmoset, as well as in the rat, whereas the serotype-9 vector resulted in contrasting effects in the two species. Neither AAV6 nor AAV9 resulted in DRG toxicity when administered via the sciatic nerve, suggesting this as a safer route of sensory nerve transduction than the currently used intrathecal or intravenous administrative routes. Furthermore, the AAV6 vector could be an optimal serotype for gene therapy for human chronic pain that has a minimal effect on other somatosensory functions of DRG neurons.
first_indexed 2024-12-14T08:11:50Z
format Article
id doaj.art-15848c6c69374436be4b3606c43fb0b6
institution Directory Open Access Journal
issn 2329-0501
language English
last_indexed 2024-12-14T08:11:50Z
publishDate 2021-12-01
publisher Elsevier
record_format Article
series Molecular Therapy: Methods & Clinical Development
spelling doaj.art-15848c6c69374436be4b3606c43fb0b62022-12-21T23:10:03ZengElsevierMolecular Therapy: Methods & Clinical Development2329-05012021-12-01231122Specific gene expression in unmyelinated dorsal root ganglion neurons in nonhuman primates by intra-nerve injection of AAV 6 vectorMoeko Kudo0Sidikejiang Wupuer1Maki Fujiwara2Yuko Saito3Shinji Kubota4Ken-ichi Inoue5Masahiko Takada6Kazuhiko Seki7Department of Neurophysiology, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Tokyo, JapanDepartment of Neurophysiology, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Tokyo, JapanSystems Neuroscience Section, Department of Neuroscience, Primate Research Institute, Kyoto University, Inuyama, Aichi, JapanDepartment of Neuropathology, Tokyo Metropolitan Institute of Gerontology, Itabashi, Tokyo, JapanDepartment of Neurophysiology, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Tokyo, JapanSystems Neuroscience Section, Department of Neuroscience, Primate Research Institute, Kyoto University, Inuyama, Aichi, JapanSystems Neuroscience Section, Department of Neuroscience, Primate Research Institute, Kyoto University, Inuyama, Aichi, JapanDepartment of Neurophysiology, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan; Corresponding author: Kazuhiko Seki, Department of Neurophysiology, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan.Adeno-associated virus 6 (AAV6) has been proposed as a potential vector candidate for specific gene expression in pain-related dorsal root ganglion (DRG) neurons, but this has not been confirmed in nonhuman primates. The aim of our study was to analyze the transduction efficiency and target specificity of this viral vector in the common marmoset by comparing it with those in the rat. When green fluorescent protein-expressing serotype-6 vector was injected into the sciatic nerve, the efficiency of gene expression in DRG neurons was comparable in both species. We found that the serotype-6 vector was largely specific to the pain-related ganglion neurons in the marmoset, as well as in the rat, whereas the serotype-9 vector resulted in contrasting effects in the two species. Neither AAV6 nor AAV9 resulted in DRG toxicity when administered via the sciatic nerve, suggesting this as a safer route of sensory nerve transduction than the currently used intrathecal or intravenous administrative routes. Furthermore, the AAV6 vector could be an optimal serotype for gene therapy for human chronic pain that has a minimal effect on other somatosensory functions of DRG neurons.http://www.sciencedirect.com/science/article/pii/S2329050121001261marmosetratAAV6AAV9DRG afferentssomatosensory
spellingShingle Moeko Kudo
Sidikejiang Wupuer
Maki Fujiwara
Yuko Saito
Shinji Kubota
Ken-ichi Inoue
Masahiko Takada
Kazuhiko Seki
Specific gene expression in unmyelinated dorsal root ganglion neurons in nonhuman primates by intra-nerve injection of AAV 6 vector
Molecular Therapy: Methods & Clinical Development
marmoset
rat
AAV6
AAV9
DRG afferents
somatosensory
title Specific gene expression in unmyelinated dorsal root ganglion neurons in nonhuman primates by intra-nerve injection of AAV 6 vector
title_full Specific gene expression in unmyelinated dorsal root ganglion neurons in nonhuman primates by intra-nerve injection of AAV 6 vector
title_fullStr Specific gene expression in unmyelinated dorsal root ganglion neurons in nonhuman primates by intra-nerve injection of AAV 6 vector
title_full_unstemmed Specific gene expression in unmyelinated dorsal root ganglion neurons in nonhuman primates by intra-nerve injection of AAV 6 vector
title_short Specific gene expression in unmyelinated dorsal root ganglion neurons in nonhuman primates by intra-nerve injection of AAV 6 vector
title_sort specific gene expression in unmyelinated dorsal root ganglion neurons in nonhuman primates by intra nerve injection of aav 6 vector
topic marmoset
rat
AAV6
AAV9
DRG afferents
somatosensory
url http://www.sciencedirect.com/science/article/pii/S2329050121001261
work_keys_str_mv AT moekokudo specificgeneexpressioninunmyelinateddorsalrootganglionneuronsinnonhumanprimatesbyintranerveinjectionofaav6vector
AT sidikejiangwupuer specificgeneexpressioninunmyelinateddorsalrootganglionneuronsinnonhumanprimatesbyintranerveinjectionofaav6vector
AT makifujiwara specificgeneexpressioninunmyelinateddorsalrootganglionneuronsinnonhumanprimatesbyintranerveinjectionofaav6vector
AT yukosaito specificgeneexpressioninunmyelinateddorsalrootganglionneuronsinnonhumanprimatesbyintranerveinjectionofaav6vector
AT shinjikubota specificgeneexpressioninunmyelinateddorsalrootganglionneuronsinnonhumanprimatesbyintranerveinjectionofaav6vector
AT kenichiinoue specificgeneexpressioninunmyelinateddorsalrootganglionneuronsinnonhumanprimatesbyintranerveinjectionofaav6vector
AT masahikotakada specificgeneexpressioninunmyelinateddorsalrootganglionneuronsinnonhumanprimatesbyintranerveinjectionofaav6vector
AT kazuhikoseki specificgeneexpressioninunmyelinateddorsalrootganglionneuronsinnonhumanprimatesbyintranerveinjectionofaav6vector