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

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Phase Ib Safety, Two-Dose Study of MultiGeneAngio in Patients with Chronic Critical Limb Ischemia. 多基因血管治疗慢性重度肢体缺血的Ib期安全性、双剂量研究。
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2017-03-01 DOI: 10.1016/j.ymthe.2016.12.019
M. Flugelman, M. Halak, B. Yoffe, J. Schneiderman, Chen Rubinstein, A. Bloom, E. Weinmann, I. Goldin, V. Ginzburg, O. Mayzler, A. Hoffman, B. Koren, D. Gershtein, M. Inbar, Marina Hutoran, Adili Tsaba
{"title":"Phase Ib Safety, Two-Dose Study of MultiGeneAngio in Patients with Chronic Critical Limb Ischemia.","authors":"M. Flugelman, M. Halak, B. Yoffe, J. Schneiderman, Chen Rubinstein, A. Bloom, E. Weinmann, I. Goldin, V. Ginzburg, O. Mayzler, A. Hoffman, B. Koren, D. Gershtein, M. Inbar, Marina Hutoran, Adili Tsaba","doi":"10.1016/j.ymthe.2016.12.019","DOIUrl":"https://doi.org/10.1016/j.ymthe.2016.12.019","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"14 1","pages":"816-825"},"PeriodicalIF":0.0,"publicationDate":"2017-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"91145909","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
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 Epub Date: 2017-01-28 DOI: 10.1016/j.ymthe.2016.12.023
Malika Hale, Taylor Mesojednik, Guillermo S Romano Ibarra, Jaya Sahni, Alison Bernard, Karen Sommer, Andrew M Scharenberg, David J Rawlings, Thor A Wagner
{"title":"Engineering HIV-Resistant, Anti-HIV Chimeric Antigen Receptor T Cells.","authors":"Malika Hale, Taylor Mesojednik, Guillermo S Romano Ibarra, Jaya Sahni, Alison Bernard, Karen Sommer, Andrew M Scharenberg, David J Rawlings, Thor A Wagner","doi":"10.1016/j.ymthe.2016.12.023","DOIUrl":"10.1016/j.ymthe.2016.12.023","url":null,"abstract":"<p><p>The treatment or cure of HIV infection by cell and gene therapy has been a goal for decades. Recent advances in both gene editing and chimeric antigen receptor (CAR) technology have created new therapeutic possibilities for a variety of diseases. Broadly neutralizing monoclonal antibodies (bNAbs) with specificity for the HIV envelope glycoprotein provide a promising means of targeting HIV-infected cells. Here we show that primary human T cells engineered to express anti-HIV CARs based on bNAbs (HIVCAR) show specific activation and killing of HIV-infected versus uninfected cells in the absence of HIV replication. We also show that homology-directed recombination of the HIVCAR gene expression cassette into the CCR5 locus enhances suppression of replicating virus compared with HIVCAR expression alone. This work demonstrates that HIV immunotherapy utilizing potent bNAb-based single-chain variable fragments fused to second-generation CAR signaling domains, delivered directly into the CCR5 locus of T cells by homology-directed gene editing, is feasible and effective. This strategy has the potential to target HIV-infected cells in HIV-infected individuals, which might help in the effort to cure HIV.</p>","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":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5363191/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"78733035","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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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