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J-GLOBAL ID：201702233852872634   整理番号：17A1430390

ヒト造血幹細胞/前駆細胞におけるCRISPR/Cas9媒介CCR5アブレーションin vivo HIV-1抵抗性を付与する【Powered by NICT】

CRISPR/Cas9-Mediated CCR5 Ablation in Human Hematopoietic Stem/Progenitor Cells Confers HIV-1 Resistance In Vivo

著者 (36件)： Xu Lei (Department of Hematopoietic Stem Cell Transplantation, 307 Hospital of PLA, Beijing 100071, China) ,  Xu Lei (Cell and Gene Therapy Center, Academy of Military Medical Sciences, Beijing 100850, China) ,  Yang Huan (Department of Cell Biology, School of Basic Medical Sciences, Peking University Stem Cell Research Center; State Key Laboratory of Natural and Biomimetic Drugs, Peking University Health Science Center, Peking University, Beijing 100191, China) ,  Yang Huan (MOE Key Laboratory of Cell Proliferation and Differentiation, College of Life Sciences, Peking-Tsinghua Center for Life Sciences, Peking University, Beijing 100191, China) ,  Yang Huan (Shenzhen Stem Cell Engineering Laboratory, Key Laboratory of Chemical Genomics, Peking University Shenzhen Graduate School, Shenzhen 518055, China) ,  Gao Yang (Department of Hematopoietic Stem Cell Transplantation, 307 Hospital of PLA, Beijing 100071, China) ,  Gao Yang (Cell and Gene Therapy Center, Academy of Military Medical Sciences, Beijing 100850, China) ,  Gao Yang (Department of Hematology, Guangzhou General Hospital of Guangzhou Military Command, Guangzhou 510010, China) ,  Chen Zeyu (Department of Cell Biology, School of Basic Medical Sciences, Peking University Stem Cell Research Center; State Key Laboratory of Natural and Biomimetic Drugs, Peking University Health Science Center, Peking University, Beijing 100191, China) ,  Chen Zeyu (MOE Key Laboratory of Cell Proliferation and Differentiation, College of Life Sciences, Peking-Tsinghua Center for Life Sciences, Peking University, Beijing 100191, China) ,  Xie Liangfu (Department of Cell Biology, School of Basic Medical Sciences, Peking University Stem Cell Research Center; State Key Laboratory of Natural and Biomimetic Drugs, Peking University Health Science Center, Peking University, Beijing 100191, China) ,  Xie Liangfu (MOE Key Laboratory of Cell Proliferation and Differentiation, College of Life Sciences, Peking-Tsinghua Center for Life Sciences, Peking University, Beijing 100191, China) ,  Liu Yulin (Department of Cell Biology, School of Basic Medical Sciences, Peking University Stem Cell Research Center; State Key Laboratory of Natural and Biomimetic Drugs, Peking University Health Science Center, Peking University, Beijing 100191, China) ,  Liu Yulin (MOE Key Laboratory of Cell Proliferation and Differentiation, College of Life Sciences, Peking-Tsinghua Center for Life Sciences, Peking University, Beijing 100191, China) ,  Liu Ying (National Center for AIDS/STD Control and Prevention, China CDC, Beijing 102206, China) ,  Wang Xiaobao (Department of Cell Biology, School of Basic Medical Sciences, Peking University Stem Cell Research Center; State Key Laboratory of Natural and Biomimetic Drugs, Peking University Health Science Center, Peking University, Beijing 100191, China) ,  Wang Xiaobao (MOE Key Laboratory of Cell Proliferation and Differentiation, College of Life Sciences, Peking-Tsinghua Center for Life Sciences, Peking University, Beijing 100191, China) ,  Li Hanwei (Department of Cell Biology, School of Basic Medical Sciences, Peking University Stem Cell Research Center; State Key Laboratory of Natural and Biomimetic Drugs, Peking University Health Science Center, Peking University, Beijing 100191, China) ,  Li Hanwei (MOE Key Laboratory of Cell Proliferation and Differentiation, College of Life Sciences, Peking-Tsinghua Center for Life Sciences, Peking University, Beijing 100191, China) ,  Lai Weifeng (Department of Cell Biology, School of Basic Medical Sciences, Peking University Stem Cell Research Center; State Key Laboratory of Natural and Biomimetic Drugs, Peking University Health Science Center, Peking University, Beijing 100191, China) ,  Lai Weifeng (MOE Key Laboratory of Cell Proliferation and Differentiation, College of Life Sciences, Peking-Tsinghua Center for Life Sciences, Peking University, Beijing 100191, China) ,  He Yuan (Department of Cell Biology, School of Basic Medical Sciences, Peking University Stem Cell Research Center; State Key Laboratory of Natural and Biomimetic Drugs, Peking University Health Science Center, Peking University, Beijing 100191, China) ,  He Yuan (MOE Key Laboratory of Cell Proliferation and Differentiation, College of Life Sciences, Peking-Tsinghua Center for Life Sciences, Peking University, Beijing 100191, China) ,  Yao Anzhi (Department of Cell Biology, School of Basic Medical Sciences, Peking University Stem Cell Research Center; State Key Laboratory of Natural and Biomimetic Drugs, Peking University Health Science Center, Peking University, Beijing 100191, China) ,  Yao Anzhi (MOE Key Laboratory of Cell Proliferation and Differentiation, College of Life Sciences, Peking-Tsinghua Center for Life Sciences, Peking University, Beijing 100191, China) ,  Ma Liying (National Center for AIDS/STD Control and Prevention, China CDC, Beijing 102206, China) ,  Shao Yiming (National Center for AIDS/STD Control and Prevention, China CDC, Beijing 102206, China) ,  Zhang Bin (Department of Hematopoietic Stem Cell Transplantation, 307 Hospital of PLA, Beijing 100071, China) ,  Zhang Bin (Cell and Gene Therapy Center, Academy of Military Medical Sciences, Beijing 100850, China) ,  Wang Chengyan (Department of Cell Biology, School of Basic Medical Sciences, Peking University Stem Cell Research Center; State Key Laboratory of Natural and Biomimetic Drugs, Peking University Health Science Center, Peking University, Beijing 100191, China) ,  Wang Chengyan (MOE Key Laboratory of Cell Proliferation and Differentiation, College of Life Sciences, Peking-Tsinghua Center for Life Sciences, Peking University, Beijing 100191, China) ,  Chen Hu (Department of Hematopoietic Stem Cell Transplantation, 307 Hospital of PLA, Beijing 100071, China) ,  Chen Hu (Cell and Gene Therapy Center, Academy of Military Medical Sciences, Beijing 100850, China) ,  Deng Hongkui (Department of Cell Biology, School of Basic Medical Sciences, Peking University Stem Cell Research Center; State Key Laboratory of Natural and Biomimetic Drugs, Peking University Health Science Center, Peking University, Beijing 100191, China) ,  Deng Hongkui (MOE Key Laboratory of Cell Proliferation and Differentiation, College of Life Sciences, Peking-Tsinghua Center for Life Sciences, Peking University, Beijing 100191, China) ,  Deng Hongkui (Shenzhen Stem Cell Engineering Laboratory, Key Laboratory of Chemical Genomics, Peking University Shenzhen Graduate School, Shenzhen 518055, China)
資料名： Molecular Therapy  (Molecular Therapy)
巻： 25  号： 8  ページ： 1782-1789  発行年： 2017年 
JST資料番号： W1762A  ISSN： 1525-0016  資料種別： 逐次刊行物 (A)
記事区分： 原著論文  発行国： オランダ (NLD)  言語： 英語 (EN)

抄録/ポイント： 天然CCR5変異を有する造血幹細胞(HSC)の移植は,患者における検出可能なHIV-1の損失を付与し,HSCにおけるCCR5遺伝子のアブレーションをHIV-1治療のための理想的な治療法。CCR5破壊はCD4~+T細胞と造血幹/前駆細胞(H SPC)の試みられてきたが,高い特異性と長期治療の可能性のある効率的な遺伝子エディティングは臨床翻訳に対する主要な課題である。ここでは,ヒトCD34~+HSPCにおけるCRISPR/Cas9遺伝子編集システムを確立し,長期再構成されたNOD/Prkdc~scid/IL 2IL-2Rγ~ヌルマウスで評価し効率的なCCR5除去を達成した。本システムにおけるCCR5破壊効率は二次移植再増殖造血細胞におけるロバストなままであった。より重要なことは,ウイルス滴定の有意な減少とヒトCD4~+T細胞の濃縮によって示されるように観察されたHIV-1抵抗効果。,長期H SCにおけるCRISPR/Cas9媒介CCR5アブレーションシステム,in vivoでのHIV 1耐性を付与することを成功裏に確立した。著者らの研究は,HIV治療のためのCCR5遺伝子編集されたHSC移植を臨床への証拠を提供する。Copyright 2017 Elsevier B.V., Amsterdam. All rights reserved. Translated from English into Japanese by JST.【Powered by NICT】

シソーラス用語： 患者 ,  遺伝子療法 ,  *造血 ,  *HIV1 ,  遺伝子 ,  *ヒト ,  変異 ,  *HIV【ウイルス】 ,  *造血幹細胞 ,  ロバスト性 ,  再構成
準シソーラス用語： *Cas9 ,  特異性 ,  *CCR5受容体 ,  *前駆細胞 , 【Automatic Indexing@JST】

著者キーワード (6件)： CCR5 ,  遺伝子編集 ,  HIV-1/AIDS ,  CRISPR ,  造血幹細胞 ,  遺伝子治療

分類 (2件)： 造血系  (EJ07020Y) ,  細胞生理一般  (EF02010S)

タイトルに関連する用語 (9件)： ヒト ,  造血幹細胞 ,  前駆細胞 ,  CRISPR ,  Cas9 ,  CCR5 ,  アブレーション ,  HIV-1 ,  抵抗性