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Efficacy of oligodendrocyte precursor cells as delivery vehicles for single-chain variable fragment to misfolded SOD1 in ALS rat model
http://hdl.handle.net/10422/00013533
http://hdl.handle.net/10422/0001353380ff99bb-89dd-447e-a796-2c2184bd8ae7
| 名前 / ファイル | ライセンス | アクション |
|---|---|---|
j.omtm.2023.01.008 (5.0 MB)
|
This is an open access article under the CC BY license
(http://creativecommons.org/licenses/by/4.0/). |
| Item type | 学術雑誌論文 / Journal Article(1) | |||||
|---|---|---|---|---|---|---|
| 公開日 | 2023-04-13 | |||||
| タイトル | ||||||
| タイトル | Efficacy of oligodendrocyte precursor cells as delivery vehicles for single-chain variable fragment to misfolded SOD1 in ALS rat model | |||||
| 言語 | ||||||
| 言語 | eng | |||||
| キーワード | ||||||
| 言語 | en | |||||
| 主題Scheme | Other | |||||
| 主題 | amyotrophic lateral sclerosis (ALS) | |||||
| キーワード | ||||||
| 言語 | en | |||||
| 主題Scheme | Other | |||||
| 主題 | superoxide dismutase 1 (SOD1) | |||||
| キーワード | ||||||
| 言語 | en | |||||
| 主題Scheme | Other | |||||
| 主題 | viral vector | |||||
| キーワード | ||||||
| 言語 | en | |||||
| 主題Scheme | Other | |||||
| 主題 | single-chain variable fragments (scFv) | |||||
| キーワード | ||||||
| 言語 | en | |||||
| 主題Scheme | Other | |||||
| 主題 | oligodendrocyte precursor cells | |||||
| キーワード | ||||||
| 言語 | en | |||||
| 主題Scheme | Other | |||||
| 主題 | transplantation | |||||
| 資源タイプ | ||||||
| 資源タイプ識別子 | http://purl.org/coar/resource_type/c_6501 | |||||
| 資源タイプ | journal article | |||||
| 著者 |
MINAMIYAMA, Sumio
× MINAMIYAMA, Sumio× SAKAI, Madoka× YAMAGUCHI, Yuko× KUSUI, Makiko× WADA, Hideki× Hikiami, Ryota× TAMAKI, Yoshitaka× ASADA-UTSUGI, Megumi× SHODAI, Akemi× MAKINO, Akiko× FUJIWARA, Noriko× AYAKI, Takashi× MAKI, Takakuni× WARITA, Hitoshi× AOKI, Masashi× TOMONAGA, Keizo× Takahashi, Ryosuke× URUSHITANI, Makoto |
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| 著者別名 |
南山, 素三雄
× 南山, 素三雄× 楠井, 真季子× 和田, 英貴× 引網, 亮太× 玉木, 良高× 浅田, めぐみ× 小代, 明美× 漆谷, 真 |
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| 抄録 | ||||||
| 内容記述タイプ | Abstract | |||||
| 内容記述 | Superoxide dismutase1 (SOD 1) mutation is a leading cause of familial amyotrophic lateral sclerosis (ALS). Growing evidence suggests that antibody therapy against misfolded SOD1 protein can be therapeutic. However, the therapeutic effects are limited, partly because of the delivery system. Therefore, we investigated the efficacy of oligodendrocyte precursor cells (OPCs) as a drug delivery vehicle of single-chain variable fragments (scFv). Using a Borna disease virus vector that is pharmacologically removable and episomally replicable in the recipient cells, we successfully transformed wild-type OPCs to secrete scFv of a novel monoclonal antibody (D3-1), specific for misfolded SOD1. Single intrathecal injection of OPCs scFvD3-1, but not OPCs alone, significantly delayed disease onset and prolonged the lifespan of ALS rat models expressing SOD1 H46R . The effect of OPC scFvD3-1 surpassed that of a 1 month intrathecal infusion of full-length D3-1 antibody alone. scFv-secreting OPCs suppressed neuronal loss and gliosis, reduced levels of misfolded SOD1 in the spinal cord, and suppressed the transcription of inflammatory genes, including Olr1, an oxidized low-density lipoprotein receptor 1. The use of OPCs as a delivery vehicle for therapeutic antibodies is a new option for ALS in which misfolded protein and oligodendrocyte dysfunction are implicated in the pathogenesis. | |||||
| 書誌情報 |
en : Molecular Therapy - Methods and Clinical Development 巻 28, p. 312-329, 発行日 2023-02-04 |
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| 出版者 | ||||||
| 出版者 | Cell Press | |||||
| ISSN | ||||||
| 収録物識別子タイプ | ISSN | |||||
| 収録物識別子 | 2329-0501 | |||||
| PMID | ||||||
| 関連タイプ | isIdenticalTo | |||||
| 識別子タイプ | PMID | |||||
| 関連識別子 | 36874245 | |||||
| PMCID | ||||||
| 識別子タイプ | URI | |||||
| 関連識別子 | http://www.ncbi.nlm.nih.gov/pmc/articles/pmc9974989/ | |||||
| 関連名称 | PMC9974989 | |||||
| DOI | ||||||
| 関連タイプ | isIdenticalTo | |||||
| 識別子タイプ | DOI | |||||
| 関連識別子 | https://doi.org/10.1016/j.omtm.2023.01.008 | |||||
| 関連名称 | 10.1016/j.omtm.2023.01.008 | |||||
| 権利 | ||||||
| 権利情報 | © 2023 The Author(s). | |||||
| フォーマット | ||||||
| 内容記述タイプ | Other | |||||
| 内容記述 | application/pdf | |||||
| 著者版フラグ | ||||||
| 出版タイプ | VoR | |||||
| 出版タイプResource | http://purl.org/coar/version/c_970fb48d4fbd8a85 | |||||
| 資源タイプ | ||||||
| 内容記述タイプ | Other | |||||
| 内容記述 | Journal Article | |||||
