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Molecular Therapy - Methods & Clinical Development最新文献

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Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy 设计并验证用于 MPSII 造血干细胞基因疗法的 GMP 干细胞制造方案
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-05-01 DOI: 10.1016/j.omtm.2024.101271
Stuart Ellison, K. Buckland, Y. Learmonth, Victoria Day, Spandan Kalra, Lauren Howe, F. Román-Rodríguez, Jose Bonafont, Laura Booth, Rebecca Holley, Jon Smythe, Simon Jones, Adrian Thrasher, Claire Booth, Brian Bigger
{"title":"Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy","authors":"Stuart Ellison, K. Buckland, Y. Learmonth, Victoria Day, Spandan Kalra, Lauren Howe, F. Román-Rodríguez, Jose Bonafont, Laura Booth, Rebecca Holley, Jon Smythe, Simon Jones, Adrian Thrasher, Claire Booth, Brian Bigger","doi":"10.1016/j.omtm.2024.101271","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101271","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"24 11","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141142701","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
Adeno-Associated Virus Perfusion Enhanced Expression: A Commercially Scalable, High Titer, High Quality Producer Cell Line Process 腺相关病毒灌注增强表达：可商业化的高滴度、高质量生产细胞系工艺
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-05-01 DOI: 10.1016/j.omtm.2024.101266
Wei Xue, Cameron Fulco, Sha Sha, Nick Alden, Jan Panteli, Patrick Hossler, James Warren
{"title":"Adeno-Associated Virus Perfusion Enhanced Expression: A Commercially Scalable, High Titer, High Quality Producer Cell Line Process","authors":"Wei Xue, Cameron Fulco, Sha Sha, Nick Alden, Jan Panteli, Patrick Hossler, James Warren","doi":"10.1016/j.omtm.2024.101266","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101266","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"73 5","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141050755","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
SERCA2a overexpression improves muscle function in a canine Duchenne muscular dystrophy model 过表达 SERCA2a 可改善犬杜氏肌营养不良症模型的肌肉功能
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-05-01 DOI: 10.1016/j.omtm.2024.101268
Kasun Kodippili, Chady H. Hakim, Matthew J. Burke, Y. Yue, James Teixeira, Keqing Zhang, Gang Yao, Gopal J. Babu, Roland W. Herzog, Dongsheng Duan
{"title":"SERCA2a overexpression improves muscle function in a canine Duchenne muscular dystrophy model","authors":"Kasun Kodippili, Chady H. Hakim, Matthew J. Burke, Y. Yue, James Teixeira, Keqing Zhang, Gang Yao, Gopal J. Babu, Roland W. Herzog, Dongsheng Duan","doi":"10.1016/j.omtm.2024.101268","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101268","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":" 42","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141131538","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
Widespread Correction of Brain Pathology in Feline Alpha-Mannosidosis by Dose Escalation of Intracisternal AAV Vector Injection 通过体内AAV载体注射的剂量递增，广泛纠正猫α-甘露糖苷病的脑部病理变化
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-05-01 DOI: 10.1016/j.omtm.2024.101272
Jacqueline Hunter, Caitlyn M. Molony, Jessica H. Bagel, Patricia O’Donnell, Charles H. Vite, Sanjeev Chawla, Harish Poptani, John H. Wolfe
{"title":"Widespread Correction of Brain Pathology in Feline Alpha-Mannosidosis by Dose Escalation of Intracisternal AAV Vector Injection","authors":"Jacqueline Hunter, Caitlyn M. Molony, Jessica H. Bagel, Patricia O’Donnell, Charles H. Vite, Sanjeev Chawla, Harish Poptani, John H. Wolfe","doi":"10.1016/j.omtm.2024.101272","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101272","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"277 9","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141144415","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
Quasi-Perfusion Studies for Intensified Lentiviral Vector Production Using a Continuous Stable Producer Cell Line 利用连续稳定的生产细胞系强化慢病毒载体生产的准灌注研究
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-05-01 DOI: 10.1016/j.omtm.2024.101264
D. J. Stibbs, Pedro Silva Couto, Yasuhiro Takeuchi, Q.A. Rafiq, Nigel B. Jackson, A. Rayat
{"title":"Quasi-Perfusion Studies for Intensified Lentiviral Vector Production Using a Continuous Stable Producer Cell Line","authors":"D. J. Stibbs, Pedro Silva Couto, Yasuhiro Takeuchi, Q.A. Rafiq, Nigel B. Jackson, A. Rayat","doi":"10.1016/j.omtm.2024.101264","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101264","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141022848","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
Genetic Alteration of SJ293TS Cells and Modification of Serum-Free Media Enhances Lentiviral Vector Production 对 SJ293TS 细胞进行基因改造并改良无血清培养基可提高慢病毒载体的产量
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-05-01 DOI: 10.1016/j.omtm.2024.101270
Matthew Bauler, Francesca Ferrara, Brandon Lowe, Jordan A. Beard, Chris Wincek, M. Wielgosz, Jeoungeun J. Park, Na Shang, Saikat Nandy, Cai Li, Deanna M. Langfitt, Sheng Zhou, R. Throm
{"title":"Genetic Alteration of SJ293TS Cells and Modification of Serum-Free Media Enhances Lentiviral Vector Production","authors":"Matthew Bauler, Francesca Ferrara, Brandon Lowe, Jordan A. Beard, Chris Wincek, M. Wielgosz, Jeoungeun J. Park, Na Shang, Saikat Nandy, Cai Li, Deanna M. Langfitt, Sheng Zhou, R. Throm","doi":"10.1016/j.omtm.2024.101270","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101270","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"12 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141140058","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
Preclinical specificity and activity of a fully human 4-1BB expressing anti-CD19 CART therapy for treatment-resistant autoimmune disease 全人类 4-1BB 表达抗 CD19 CART疗法的临床前特异性和活性，用于治疗耐药的自身免疫性疾病
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-05-01 DOI: 10.1016/j.omtm.2024.101267
Binghao J. Peng, Andrea Alvarado, Hangameh Cassim, Soprina Guarneri, Steven Wong, Jonathan Willis, Julia SantaMaria, Ashley Martynchuk, Victoria Stratton, Darshil Patel, Chien-Chung Chen, Yan Li, Gwendolyn K. Binder, Rebecca Dryer-Minnerly, Jinmin Lee, Samik Basu
{"title":"Preclinical specificity and activity of a fully human 4-1BB expressing anti-CD19 CART therapy for treatment-resistant autoimmune disease","authors":"Binghao J. Peng, Andrea Alvarado, Hangameh Cassim, Soprina Guarneri, Steven Wong, Jonathan Willis, Julia SantaMaria, Ashley Martynchuk, Victoria Stratton, Darshil Patel, Chien-Chung Chen, Yan Li, Gwendolyn K. Binder, Rebecca Dryer-Minnerly, Jinmin Lee, Samik Basu","doi":"10.1016/j.omtm.2024.101267","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101267","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"346 8","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141143870","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 and Testing of a Versatile Genome Editing Application Reporter (V-GEAR) System 多功能基因组编辑应用报告器（V-GEAR）系统的开发与测试
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-04-01 DOI: 10.1016/j.omtm.2024.101253
Evan W. Kleinboehl, Kanut Laoharawee, Walker S. Lahr, Jacob D. Jensen, Joseph J. Peterson, Jason B. Bell, Beau R. Webber, B. Moriarity
{"title":"Development and Testing of a Versatile Genome Editing Application Reporter (V-GEAR) System","authors":"Evan W. Kleinboehl, Kanut Laoharawee, Walker S. Lahr, Jacob D. Jensen, Joseph J. Peterson, Jason B. Bell, Beau R. Webber, B. Moriarity","doi":"10.1016/j.omtm.2024.101253","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101253","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"943 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140774673","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
Rescue of myocytes and locomotion through AAV2/9-2YF intracisternal gene therapy in a rat model of creatine transporter deficiency 在肌酸转运体缺乏症大鼠模型中，通过 AAV2/9-2YF 内部基因疗法挽救肌细胞和运动能力
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-04-01 DOI: 10.1016/j.omtm.2024.101251
Gabriella Fernandes-Pires, Marcelo Duarte Azevedo, Marc Lanzillo, Clothilde Roux-Petronelli, P. Binz, Cristina Cudalbu, Carmen Sandi, Liliane Tenenbaum, Olivier Braissant
{"title":"Rescue of myocytes and locomotion through AAV2/9-2YF intracisternal gene therapy in a rat model of creatine transporter deficiency","authors":"Gabriella Fernandes-Pires, Marcelo Duarte Azevedo, Marc Lanzillo, Clothilde Roux-Petronelli, P. Binz, Cristina Cudalbu, Carmen Sandi, Liliane Tenenbaum, Olivier Braissant","doi":"10.1016/j.omtm.2024.101251","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101251","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"432 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140782346","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
A Novel High-Titer, Bifunctional Lentiviral Vector for Autologous Hematopoietic Stem Cell Gene Therapy of Sickle Cell Disease 用于镰状细胞病自体造血干细胞基因治疗的新型高滴度双功能慢病毒载体
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-04-01 DOI: 10.1016/j.omtm.2024.101254
Kevyn L. Hart, Boya Liu, Devin Brown, Beatriz Campo-Fernández, Kevin Tam, Katherine Orr, R. Hollis, Christian Brendel, David A. Williams, Donald B. Kohn
{"title":"A Novel High-Titer, Bifunctional Lentiviral Vector for Autologous Hematopoietic Stem Cell Gene Therapy of Sickle Cell Disease","authors":"Kevyn L. Hart, Boya Liu, Devin Brown, Beatriz Campo-Fernández, Kevin Tam, Katherine Orr, R. Hollis, Christian Brendel, David A. Williams, Donald B. Kohn","doi":"10.1016/j.omtm.2024.101254","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101254","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"1216 7","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140774194","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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