Pub Date : 2023-02-24DOI: 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
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.
{"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}
Pub Date : 2022-06-01DOI: 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}
Pub Date : 2022-06-01DOI: 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}
Pub Date : 2022-05-01DOI: 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}
Pub Date : 2022-03-01DOI: 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}
Pub Date : 2021-04-24DOI: 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}
Pub Date : 2021-03-16DOI: 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}
Pub Date : 2020-10-07DOI: 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}
Pub Date : 2020-09-02DOI: 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}
Pub Date : 2020-08-05DOI: 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}
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