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Molecular Therapy: the Journal of the American Society of Gene Therapy最新文献

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Inducible Caspase-9 Selectively Modulates the Toxicities of CD19-Specific Chimeric Antigen Receptor-Modified T Cells. 诱导型Caspase-9选择性调节cd19特异性嵌合抗原受体修饰的T细胞的毒性
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2017-03-01 DOI: 10.1016/j.ymthe.2017.01.011
I. Diaconu, B. Ballard, Ming Zhang, Yuhui Chen, J. West, G. Dotti, B. Savoldo
{"title":"Inducible Caspase-9 Selectively Modulates the Toxicities of CD19-Specific Chimeric Antigen Receptor-Modified T Cells.","authors":"I. Diaconu, B. Ballard, Ming Zhang, Yuhui Chen, J. West, G. Dotti, B. Savoldo","doi":"10.1016/j.ymthe.2017.01.011","DOIUrl":"https://doi.org/10.1016/j.ymthe.2017.01.011","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"1 1","pages":"580-592"},"PeriodicalIF":0.0,"publicationDate":"2017-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"77226920","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 180
Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery. 在IV rAAV9-hNAGLU基因递送后，MPS IIIB小鼠的代谢组学损伤几乎完全纠正，相应的功能获益
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2017-03-01 DOI: 10.1016/j.ymthe.2016.12.025
H. Fu, Aaron S. Meadows, Tierra A Ware, R. Mohney, D. Mccarty
{"title":"Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery.","authors":"H. Fu, Aaron S. Meadows, Tierra A Ware, R. Mohney, D. Mccarty","doi":"10.1016/j.ymthe.2016.12.025","DOIUrl":"https://doi.org/10.1016/j.ymthe.2016.12.025","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"22 1","pages":"792-802"},"PeriodicalIF":0.0,"publicationDate":"2017-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"82031861","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 15
Pericardial Fluid Exosomes: A New Material to Treat Cardiovascular Disease. 心包液外泌体:治疗心血管疾病的新材料
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2017-03-01 DOI: 10.1016/j.ymthe.2017.02.002
S. Sahoo, P. Mathiyalagan, R. Hajjar
{"title":"Pericardial Fluid Exosomes: A New Material to Treat Cardiovascular Disease.","authors":"S. Sahoo, P. Mathiyalagan, R. Hajjar","doi":"10.1016/j.ymthe.2017.02.002","DOIUrl":"https://doi.org/10.1016/j.ymthe.2017.02.002","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"20 1","pages":"568-569"},"PeriodicalIF":0.0,"publicationDate":"2017-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"82095820","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 21
Engineering HIV-Resistant, Anti-HIV Chimeric Antigen Receptor T Cells. 工程抗hiv，抗hiv嵌合抗原受体T细胞。
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2017-03-01 DOI: 10.1016/j.ymthe.2016.12.023
Malika Hale, Taylor D. Mesojednik, G. S. Romano Ibarra, J. Sahni, Alison Bernard, Karen Sommer, A. Scharenberg, D. Rawlings, Thor A Wagner
{"title":"Engineering HIV-Resistant, Anti-HIV Chimeric Antigen Receptor T Cells.","authors":"Malika Hale, Taylor D. Mesojednik, G. S. Romano Ibarra, J. Sahni, Alison Bernard, Karen Sommer, A. Scharenberg, D. Rawlings, Thor A Wagner","doi":"10.1016/j.ymthe.2016.12.023","DOIUrl":"https://doi.org/10.1016/j.ymthe.2016.12.023","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"60 1","pages":"570-579"},"PeriodicalIF":0.0,"publicationDate":"2017-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"78733035","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 129
miR-18a-5p Inhibits Sub-pleural Pulmonary Fibrosis by Targeting TGF-β Receptor II. miR-18a-5p靶向TGF-β受体II抑制胸膜下肺纤维化
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2017-03-01 DOI: 10.1016/j.ymthe.2016.12.017
Qian Zhang, Hong Ye, Fei-xiang Xiang, Lin-Jie Song, Li-Ling Zhou, P. Cai, Jian-chu Zhang, Fan Yu, Huanzhong Shi, Yunchao Su, J. Xin, Wan-Li Ma
{"title":"miR-18a-5p Inhibits Sub-pleural Pulmonary Fibrosis by Targeting TGF-β Receptor II.","authors":"Qian Zhang, Hong Ye, Fei-xiang Xiang, Lin-Jie Song, Li-Ling Zhou, P. Cai, Jian-chu Zhang, Fan Yu, Huanzhong Shi, Yunchao Su, J. Xin, Wan-Li Ma","doi":"10.1016/j.ymthe.2016.12.017","DOIUrl":"https://doi.org/10.1016/j.ymthe.2016.12.017","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"93 1","pages":"728-738"},"PeriodicalIF":0.0,"publicationDate":"2017-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"83863644","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 55
Targeting the HDAC2/HNF-4A/miR-101b/AMPK Pathway Rescues Tauopathy and Dendritic Abnormalities in Alzheimer's Disease. 靶向HDAC2/HNF-4A/miR-101b/AMPK通路拯救阿尔茨海默病的tau病和树突状异常
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2017-03-01 DOI: 10.1016/j.ymthe.2017.01.018
Dan Liu, Hui Tang, Xinyan Li, Man-Fei Deng, N. Wei, Xiong Wang, Yafan Zhou, Ding-Qi Wang, Peng Fu, Jian-Zhi Wang, Sébastien S. Hébert, Jian-Guo Chen, Youming Lu, Ling-Qiang Zhu
{"title":"Targeting the HDAC2/HNF-4A/miR-101b/AMPK Pathway Rescues Tauopathy and Dendritic Abnormalities in Alzheimer's Disease.","authors":"Dan Liu, Hui Tang, Xinyan Li, Man-Fei Deng, N. Wei, Xiong Wang, Yafan Zhou, Ding-Qi Wang, Peng Fu, Jian-Zhi Wang, Sébastien S. Hébert, Jian-Guo Chen, Youming Lu, Ling-Qiang Zhu","doi":"10.1016/j.ymthe.2017.01.018","DOIUrl":"https://doi.org/10.1016/j.ymthe.2017.01.018","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"29 1","pages":"752-764"},"PeriodicalIF":0.0,"publicationDate":"2017-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"82299713","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 76
Antisense Reduction of Mutant COMP Reduces Growth Plate Chondrocyte Pathology. 突变体COMP的反义减少减少生长板软骨细胞病理。
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2017-03-01 DOI: 10.1016/j.ymthe.2016.12.024
K. Posey, F. Coustry, Alka C. Veerisetty, Mohammad G. Hossain, D. Gattis, S. Booten, J. Alcorn, P. Seth, J. Hecht
{"title":"Antisense Reduction of Mutant COMP Reduces Growth Plate Chondrocyte Pathology.","authors":"K. Posey, F. Coustry, Alka C. Veerisetty, Mohammad G. Hossain, D. Gattis, S. Booten, J. Alcorn, P. Seth, J. Hecht","doi":"10.1016/j.ymthe.2016.12.024","DOIUrl":"https://doi.org/10.1016/j.ymthe.2016.12.024","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"38 1","pages":"705-714"},"PeriodicalIF":0.0,"publicationDate":"2017-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"78419428","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 15
Involvement of a Rac1-Dependent Macropinocytosis Pathway in Plasmid DNA Delivery by Electrotransfection. 电转染介导的质粒DNA递送中rac1依赖性巨噬细胞增多途径的参与。
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2017-03-01 DOI: 10.1016/j.ymthe.2016.12.009
Mao Mao, Liangli Wang, Chun-Chi Chang, Katheryn E. Rothenberg, Jianyong Huang, Yingxiao Wang, B. Hoffman, P. Liton, F. Yuan
{"title":"Involvement of a Rac1-Dependent Macropinocytosis Pathway in Plasmid DNA Delivery by Electrotransfection.","authors":"Mao Mao, Liangli Wang, Chun-Chi Chang, Katheryn E. Rothenberg, Jianyong Huang, Yingxiao Wang, B. Hoffman, P. Liton, F. Yuan","doi":"10.1016/j.ymthe.2016.12.009","DOIUrl":"https://doi.org/10.1016/j.ymthe.2016.12.009","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"96 1","pages":"803-815"},"PeriodicalIF":0.0,"publicationDate":"2017-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"90326402","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 29
Manipulating PML SUMOylation via Silencing UBC9 and RNF4 Regulates Cardiac Fibrosis. 通过沉默UBC9和RNF4调控PML summoylation调节心脏纤维化。
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2017-03-01 DOI: 10.1016/j.ymthe.2016.12.021
Yu Liu, Dan Zhao, Fang Qiu, Lingling Zhang, Shang-Kun Liu, Yuan-Yuan Li, Mei-Tong Liu, Di Wu, Jia-Xin Wang, Xiao-Qing Ding, Yanxin Liu, Chang-Jiang Dong, Xiaoqi Shao, Bao‐feng Yang, Wen-Feng Chu
{"title":"Manipulating PML SUMOylation via Silencing UBC9 and RNF4 Regulates Cardiac Fibrosis.","authors":"Yu Liu, Dan Zhao, Fang Qiu, Lingling Zhang, Shang-Kun Liu, Yuan-Yuan Li, Mei-Tong Liu, Di Wu, Jia-Xin Wang, Xiao-Qing Ding, Yanxin Liu, Chang-Jiang Dong, Xiaoqi Shao, Bao‐feng Yang, Wen-Feng Chu","doi":"10.1016/j.ymthe.2016.12.021","DOIUrl":"https://doi.org/10.1016/j.ymthe.2016.12.021","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"119 1","pages":"666-678"},"PeriodicalIF":0.0,"publicationDate":"2017-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"91348313","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 43
Partial Restoration of CFTR Function in cftr-Null Mice following Targeted Cell Replacement Therapy. 靶向细胞替代治疗后CFTR缺失小鼠CFTR功能部分恢复
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2017-03-01 DOI: 10.1016/j.ymthe.2016.11.018
P. Duchesneau, R. Besla, M. Derouet, Li Guo, G. Karoubi, A. Silberberg, A. Wong, T. Waddell
{"title":"Partial Restoration of CFTR Function in cftr-Null Mice following Targeted Cell Replacement Therapy.","authors":"P. Duchesneau, R. Besla, M. Derouet, Li Guo, G. Karoubi, A. Silberberg, A. Wong, T. Waddell","doi":"10.1016/j.ymthe.2016.11.018","DOIUrl":"https://doi.org/10.1016/j.ymthe.2016.11.018","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"109 1","pages":"654-665"},"PeriodicalIF":0.0,"publicationDate":"2017-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"85721334","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 16
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