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Molecular Therapy - Methods & Clinical Development最新文献

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CAR-T cell manufacturing landscape – lessons from the past decade and considerations for early clinical development CAR-T 细胞生产现状--过去十年的经验教训和早期临床开发的注意事项
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-04-01 DOI: 10.1016/j.omtm.2024.101250
Juliana Dias, John Garcia, Giulia Agliardi, Claire Roddie
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引用次数: 0
An HPLC-SEC-based rapid quantification method for vesicular stomatitis virus particles to facilitate process development 基于 HPLC-SEC 的水泡性口炎病毒颗粒快速定量方法促进工艺开发
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-04-01 DOI: 10.1016/j.omtm.2024.101252
Adrian Schimek, Judy KM. Ng, Ioannes Basbas, Fabian Martin, Dongyue Xin, David Saleh, Jürgen Hubbuch
{"title":"An HPLC-SEC-based rapid quantification method for vesicular stomatitis virus particles to facilitate process development","authors":"Adrian Schimek, Judy KM. Ng, Ioannes Basbas, Fabian Martin, Dongyue Xin, David Saleh, Jürgen Hubbuch","doi":"10.1016/j.omtm.2024.101252","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101252","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"1107 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140774112","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
Glycosylation of recombinant adeno-associated virus serotype 6 重组腺相关病毒血清 6 型的糖基化
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-04-01 DOI: 10.1016/j.omtm.2024.101256
Yuki Yamaguchi, Kentaro Ishii, Sachiko Koizumi, Hiroaki Sakaue, Takahiro Maruno, Mitsuko Fukuhara, Risa Shibuya, Yasuo Tsunaka, Aoba Matsushita, Karin Bandoh, Tetsuo Torisu, Chie Murata-Kishimoto, Azusa Tomioka, Saho Mizukado, Hiroyuki Kaji, Yuji Kashiwakura, Tsukasa Ohmori, Atsushi Kuno, Susumu Uchiyama
{"title":"Glycosylation of recombinant adeno-associated virus serotype 6","authors":"Yuki Yamaguchi, Kentaro Ishii, Sachiko Koizumi, Hiroaki Sakaue, Takahiro Maruno, Mitsuko Fukuhara, Risa Shibuya, Yasuo Tsunaka, Aoba Matsushita, Karin Bandoh, Tetsuo Torisu, Chie Murata-Kishimoto, Azusa Tomioka, Saho Mizukado, Hiroyuki Kaji, Yuji Kashiwakura, Tsukasa Ohmori, Atsushi Kuno, Susumu Uchiyama","doi":"10.1016/j.omtm.2024.101256","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101256","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"212 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140777584","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
mtDNA Analysis using Mitopore 利用 Mitopore 进行 mtDNA 分析
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-03-01 DOI: 10.1016/j.omtm.2024.101231
J. Dobner, T. Nguyen, M.G. Pavez-Giani, L. Cyganek, F. Distelmaier, J. Krutmann, A. Prigione, A. Rossi
{"title":"mtDNA Analysis using Mitopore","authors":"J. Dobner, T. Nguyen, M.G. Pavez-Giani, L. Cyganek, F. Distelmaier, J. Krutmann, A. Prigione, A. Rossi","doi":"10.1016/j.omtm.2024.101231","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101231","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"16 18","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140278035","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
Measurement solutions and standards for advanced therapy 先进疗法的测量解决方案和标准
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-03-01 DOI: 10.1016/j.omtm.2024.101219
Lili Wang, Samantha Maragh, Edward J Kwee, Jamie L. Almeida, Sierra D Miller, Tara Eskandari, Sheng Lin-Gibson
{"title":"Measurement solutions and standards for advanced therapy","authors":"Lili Wang, Samantha Maragh, Edward J Kwee, Jamie L. Almeida, Sierra D Miller, Tara Eskandari, Sheng Lin-Gibson","doi":"10.1016/j.omtm.2024.101219","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101219","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"108 27","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140088595","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
Protein Phosphatase 2A Anchoring Disruptor Gene Therapy for Familial Dilated Cardiomyopathy 蛋白磷酸酶 2A 锚定干扰基因疗法治疗家族性扩张型心肌病
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-03-01 DOI: 10.1016/j.omtm.2024.101233
Xueyi Li, Jinliang Li, Anne-Maj Samuelsson, Hrishikesh Thakur, M. Kapiloff
{"title":"Protein Phosphatase 2A Anchoring Disruptor Gene Therapy for Familial Dilated Cardiomyopathy","authors":"Xueyi Li, Jinliang Li, Anne-Maj Samuelsson, Hrishikesh Thakur, M. Kapiloff","doi":"10.1016/j.omtm.2024.101233","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101233","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"63 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140272153","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
Durable transgene expression and efficient re-administration after rAAV2.5T-mediated fCFTRΔR gene delivery to adult ferret lungs rAAV2.5T 介导的 fCFTRΔR 基因输送到成年雪貂肺部后的持久转基因表达和高效再给药
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-03-01 DOI: 10.1016/j.omtm.2024.101244
Yinghua Tang, M. Ebadi, J. Lei, Z. Feng, S. Fakhari, P. Wu, Mark D. Smith, M. Limberis, R. Kolbeck, K. Excoffon, Ziying Yan, J. Engelhardt
{"title":"Durable transgene expression and efficient re-administration after rAAV2.5T-mediated fCFTRΔR gene delivery to adult ferret lungs","authors":"Yinghua Tang, M. Ebadi, J. Lei, Z. Feng, S. Fakhari, P. Wu, Mark D. Smith, M. Limberis, R. Kolbeck, K. Excoffon, Ziying Yan, J. Engelhardt","doi":"10.1016/j.omtm.2024.101244","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101244","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"106 S5","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140405673","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
Development of Adenoviral vectors that transduce Purkinje cells and other cerebellar cell-types in the cerebellum of a humanized mouse model 开发能在人源化小鼠模型小脑中转导浦肯野细胞和其他小脑细胞类型的腺病毒载体
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-03-01 DOI: 10.1016/j.omtm.2024.101243
Emre Kul, Uchechi Okoroafor, Amanda Dougherty, Lauren Palkovic, Hao Li, Paula Valiño-Ramos, Leah Aberman, Samuel M. Young
{"title":"Development of Adenoviral vectors that transduce Purkinje cells and other cerebellar cell-types in the cerebellum of a humanized mouse model","authors":"Emre Kul, Uchechi Okoroafor, Amanda Dougherty, Lauren Palkovic, Hao Li, Paula Valiño-Ramos, Leah Aberman, Samuel M. Young","doi":"10.1016/j.omtm.2024.101243","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101243","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"73 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140405117","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
Gene Editing based Targeted Integration for Correction of the Wiskott-Aldrich Syndrome 基于基因编辑的靶向整合技术矫正威斯科特-阿尔德里奇综合征
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-02-01 DOI: 10.1016/j.omtm.2024.101208
Melissa Pille, John M. Avila, So Hyun Park, Cuong Q. Le, Haipeng Xue, F. Haerynck, Lavanya Saxena, Ciaran M. Lee, E. Shpall, Gang Bao, Bart Vandekerckhove, Brian R. Davis
{"title":"Gene Editing based Targeted Integration for Correction of the Wiskott-Aldrich Syndrome","authors":"Melissa Pille, John M. Avila, So Hyun Park, Cuong Q. Le, Haipeng Xue, F. Haerynck, Lavanya Saxena, Ciaran M. Lee, E. Shpall, Gang Bao, Bart Vandekerckhove, Brian R. Davis","doi":"10.1016/j.omtm.2024.101208","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101208","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"228 19","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139831908","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
Gene Editing based Targeted Integration for Correction of the Wiskott-Aldrich Syndrome 基于基因编辑的靶向整合技术矫正威斯科特-阿尔德里奇综合征
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-02-01 DOI: 10.1016/j.omtm.2024.101208
Melissa Pille, John M. Avila, So Hyun Park, Cuong Q. Le, Haipeng Xue, F. Haerynck, Lavanya Saxena, Ciaran M. Lee, E. Shpall, Gang Bao, Bart Vandekerckhove, Brian R. Davis
{"title":"Gene Editing based Targeted Integration for Correction of the Wiskott-Aldrich Syndrome","authors":"Melissa Pille, John M. Avila, So Hyun Park, Cuong Q. Le, Haipeng Xue, F. Haerynck, Lavanya Saxena, Ciaran M. Lee, E. Shpall, Gang Bao, Bart Vandekerckhove, Brian R. Davis","doi":"10.1016/j.omtm.2024.101208","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101208","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"20 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139891710","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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