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

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In Vivo CRISPR Gene Editing in Patients with Herpes Stromal Keratitis 体内CRISPR基因编辑在疱疹间质性角膜炎患者中的应用
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2023-02-24 DOI: 10.1101/2023.02.21.23285822
Anji Wei, Di Yin, Zimeng Zhai, Sikai Ling, Huangying Le, Lijia Tian, Jianjiang Xu, Soren R. Paludan, Yujia Cai, Jiaxu Hong
{"title":"In Vivo CRISPR Gene Editing in Patients with Herpes Stromal Keratitis","authors":"Anji Wei, Di Yin, Zimeng Zhai, Sikai Ling, Huangying Le, Lijia Tian, Jianjiang Xu, Soren R. Paludan, Yujia Cai, Jiaxu Hong","doi":"10.1101/2023.02.21.23285822","DOIUrl":"https://doi.org/10.1101/2023.02.21.23285822","url":null,"abstract":"In vivo CRISPR gene therapy holds large clinical potential, but the safety and efficacy remain largely unknown. Here, we injected a single dose of HSV-1-targeting CRISPR formulation in the cornea of three patients with severe refractory herpes stromal keratitis (HSK) during corneal transplantation. Our study is an investigated initiated, open-label, single-arm, non-randomized interventional trial at a single center (NCT04560790). We found neither detectable CRISPR-induced off-target cleavages by GUIDE-seq nor systemic adverse events for 18 months on average in all three patients. The HSV-1 remained undetectable during the study. Our preliminary clinical results suggest that in vivo gene editing targeting the HSV-1 genome holds acceptable safety as a potential therapy for HSK.","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"21 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2023-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"72727706","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: 3
ASGCT 2022 - Bigger than Ever. ASGCT 2022 -比以往任何时候都更大
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2022-06-01 DOI: 10.1016/j.ymthe.2022.06.009
R. Herzog
{"title":"ASGCT 2022 - Bigger than Ever.","authors":"R. Herzog","doi":"10.1016/j.ymthe.2022.06.009","DOIUrl":"https://doi.org/10.1016/j.ymthe.2022.06.009","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"144 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"89023028","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: 0
Therapy in Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa. 视紫红质介导的常染色体显性视网膜色素变性的治疗。
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2022-06-01 DOI: 10.1016/j.ymthe.2022.06.007
Da Meng, Sara D. Ragi, S. Tsang
{"title":"Therapy in Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa.","authors":"Da Meng, Sara D. Ragi, S. Tsang","doi":"10.1016/j.ymthe.2022.06.007","DOIUrl":"https://doi.org/10.1016/j.ymthe.2022.06.007","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"121 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"73030458","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: 28
Retraction Notice to: CRNDE Promotes Malignant Progression of Glioma by Attenuating miR-384/PIWIL4/STAT3 Axis. 撤回通知:CRNDE通过减弱miR-384/PIWIL4/STAT3轴促进胶质瘤的恶性进展。
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2022-05-01 DOI: 10.1016/j.ymthe.2022.05.016
Jian Zheng, Xiaobai Liu, PingWang, Yixue Xue, Jun Ma, Chengbin Qu, Yunhui Liu
{"title":"Retraction Notice to: CRNDE Promotes Malignant Progression of Glioma by Attenuating miR-384/PIWIL4/STAT3 Axis.","authors":"Jian Zheng, Xiaobai Liu, PingWang, Yixue Xue, Jun Ma, Chengbin Qu, Yunhui Liu","doi":"10.1016/j.ymthe.2022.05.016","DOIUrl":"https://doi.org/10.1016/j.ymthe.2022.05.016","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"28 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2022-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"87796182","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: 0
Retraction Notice to: The YY1-HOTAIR-MMP2 Signaling Axis Controls Trophoblast Invasion at the Maternal-Fetal Interface. YY1-HOTAIR-MMP2信号轴控制母胎界面滋养细胞侵袭
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2022-03-01 DOI: 10.1016/j.ymthe.2022.02.017
Yan Zhang, Feng Jin, Xiao-Cui Li, Fu-jin Shen, Xiao-ling Ma, Fan Wu, Siming Zhang, Weihong Zeng, Xiaorui Liu, Jianghe Fan, Yi Lin, Fu-Ju Tian
{"title":"Retraction Notice to: The YY1-HOTAIR-MMP2 Signaling Axis Controls Trophoblast Invasion at the Maternal-Fetal Interface.","authors":"Yan Zhang, Feng Jin, Xiao-Cui Li, Fu-jin Shen, Xiao-ling Ma, Fan Wu, Siming Zhang, Weihong Zeng, Xiaorui Liu, Jianghe Fan, Yi Lin, Fu-Ju Tian","doi":"10.1016/j.ymthe.2022.02.017","DOIUrl":"https://doi.org/10.1016/j.ymthe.2022.02.017","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"62 1","pages":"1352"},"PeriodicalIF":0.0,"publicationDate":"2022-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"90738399","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: 0
Mavis Agbandje-McKenna's lifelong commitment to teaching and research. Mavis Agbandje-McKenna毕生致力于教学和研究。
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2021-04-24 DOI: 10.1016/j.ymthe.2021.03.007
B. Byrne, Angela L McCall, Beverly L. Davidson, R. Samulski
{"title":"Mavis Agbandje-McKenna's lifelong commitment to teaching and research.","authors":"B. Byrne, Angela L McCall, Beverly L. Davidson, R. Samulski","doi":"10.1016/j.ymthe.2021.03.007","DOIUrl":"https://doi.org/10.1016/j.ymthe.2021.03.007","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"23 4","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"91425607","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: 0
ASGCT co-founder and past president Arthur Nienhuis (1941-2021). ASGCT联合创始人兼前总裁Arthur Nienhuis(1941-2021)。
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2021-03-16 DOI: 10.1016/j.ymthe.2021.03.006
C. Dunbar, D. Bodine
{"title":"ASGCT co-founder and past president Arthur Nienhuis (1941-2021).","authors":"C. Dunbar, D. Bodine","doi":"10.1016/j.ymthe.2021.03.006","DOIUrl":"https://doi.org/10.1016/j.ymthe.2021.03.006","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"8 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2021-03-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"88289898","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: 0
Research Highlights. 研究突出了。
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2020-10-07 DOI: 10.1016/j.ymthe.2020.09.022
{"title":"Research Highlights.","authors":"","doi":"10.1016/j.ymthe.2020.09.022","DOIUrl":"https://doi.org/10.1016/j.ymthe.2020.09.022","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"39 1","pages":"2101-2102"},"PeriodicalIF":0.0,"publicationDate":"2020-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"89547128","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: 0
Research Highlights. 研究突出了。
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2020-09-02 DOI: 10.1016/j.ymthe.2020.08.009
{"title":"Research Highlights.","authors":"","doi":"10.1016/j.ymthe.2020.08.009","DOIUrl":"https://doi.org/10.1016/j.ymthe.2020.08.009","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"7 1","pages":"1936-1937"},"PeriodicalIF":0.0,"publicationDate":"2020-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"72626179","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: 0
Research Highlights. 研究突出了。
Molecular Therapy: the Journal of the American Society of Gene Therapy Pub Date : 2020-08-05 DOI: 10.1016/j.ymthe.2020.07.013
{"title":"Research Highlights.","authors":"","doi":"10.1016/j.ymthe.2020.07.013","DOIUrl":"https://doi.org/10.1016/j.ymthe.2020.07.013","url":null,"abstract":"","PeriodicalId":19039,"journal":{"name":"Molecular Therapy: the Journal of the American Society of Gene Therapy","volume":"136 1","pages":"1747-1748"},"PeriodicalIF":0.0,"publicationDate":"2020-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"75693166","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: 0
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