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Molecular Therapy - Methods & Clinical Development最新文献

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Evaluation of a Rapid Multi-Attribute Combinatorial High-Throughput UV-Vis/DLS/SLS Analytical Platform for rAAV Quantification and Characterization 评估用于 rAAV 定量和表征的快速多属性组合高通量 UV-Vis/DLS/SLS 分析平台
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-07-01 DOI: 10.1016/j.omtm.2024.101298
Xueyuan Liu, Riffard Jean-Gilles, Julia Baginski, Christina Cai, Ruilan Yan, Lili Zhang, Kevin Lance, Johannes C.M. van der Loo, Beverly L. Davidson
{"title":"Evaluation of a Rapid Multi-Attribute Combinatorial High-Throughput UV-Vis/DLS/SLS Analytical Platform for rAAV Quantification and Characterization","authors":"Xueyuan Liu, Riffard Jean-Gilles, Julia Baginski, Christina Cai, Ruilan Yan, Lili Zhang, Kevin Lance, Johannes C.M. van der Loo, Beverly L. Davidson","doi":"10.1016/j.omtm.2024.101298","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101298","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"10 8","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141698559","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
Suppression of toxic transgene expression by optimized artificial miRNAs increases AAV vector yields in HEK-293 cells 用优化的人工 miRNA 抑制毒性转基因表达，提高 HEK-293 细胞中 AAV 载体的产量
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-06-01 DOI: 10.1016/j.omtm.2024.101280
Gina Blahetek, Christine Mayer, Johannes Zuber, Martin Lenter, Benjamin Strobel
{"title":"Suppression of toxic transgene expression by optimized artificial miRNAs increases AAV vector yields in HEK-293 cells","authors":"Gina Blahetek, Christine Mayer, Johannes Zuber, Martin Lenter, Benjamin Strobel","doi":"10.1016/j.omtm.2024.101280","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101280","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"31 6","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141397191","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
Biomanufacturing in gene and cell therapy 基因和细胞疗法中的生物制造
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-06-01 DOI: 10.1016/j.omtm.2024.101261
Daniel Stone, Xiuyang Wang, Mohamed Abou-el-Enein
{"title":"Biomanufacturing in gene and cell therapy","authors":"Daniel Stone, Xiuyang Wang, Mohamed Abou-el-Enein","doi":"10.1016/j.omtm.2024.101261","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101261","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"30 8","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141231682","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
Cardiac Gene Delivery Using Ultrasound: State of the Field 利用超声波传递心脏基因：领域现状
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-06-01 DOI: 10.1016/j.omtm.2024.101277
Davindra Singh, Elahe Memari, Stephanie He, Hossein Yusefi, Brandon Helfield
{"title":"Cardiac Gene Delivery Using Ultrasound: State of the Field","authors":"Davindra Singh, Elahe Memari, Stephanie He, Hossein Yusefi, Brandon Helfield","doi":"10.1016/j.omtm.2024.101277","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101277","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"65 6","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141408570","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
Systemic delivery of AAV-GCDH ameliorates HLD-induced phenotype in a glutaric aciduria type I mouse model 系统输送 AAV-GCDH 可改善戊二酸尿症 I 型小鼠模型中 HLD 诱导的表型
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-06-01 DOI: 10.1016/j.omtm.2024.101276
A. Mateu-Bosch, E. Segur-Bailach, Emma Muñoz-Moreno, M. Barallobre, Maria Lourdes Arbonés, S. Gea-Sorlí, F. Tort, Antonia Ribes, J. García-Villoria, C. Fillat
{"title":"Systemic delivery of AAV-GCDH ameliorates HLD-induced phenotype in a glutaric aciduria type I mouse model","authors":"A. Mateu-Bosch, E. Segur-Bailach, Emma Muñoz-Moreno, M. Barallobre, Maria Lourdes Arbonés, S. Gea-Sorlí, F. Tort, Antonia Ribes, J. García-Villoria, C. Fillat","doi":"10.1016/j.omtm.2024.101276","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101276","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"7 5","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141391930","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
Mesenchymal Stromal Cells Protect Combined Oncolytic and Helper Dependent Adenoviruses from Humoral Immunity 间充质基质细胞保护溶瘤和辅助细胞依赖性腺病毒免受体液免疫的侵袭
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-06-01 DOI: 10.1016/j.omtm.2024.101279
A. Ozcan, Arianexys Aquino Lόpez, Alexandra N. Wolff, Audrey Ma, Amanda Rosewell Shaw, Masataka Suzuki, Malcolm K. Brenner, M. McKenna
{"title":"Mesenchymal Stromal Cells Protect Combined Oncolytic and Helper Dependent Adenoviruses from Humoral Immunity","authors":"A. Ozcan, Arianexys Aquino Lόpez, Alexandra N. Wolff, Audrey Ma, Amanda Rosewell Shaw, Masataka Suzuki, Malcolm K. Brenner, M. McKenna","doi":"10.1016/j.omtm.2024.101279","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101279","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"15 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141400038","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
The postnatal injection of AAV9-FOXG1 rescues corpus callosum agenesis and other brain deficits in the mouse model of FOXG1 syndrome 出生后注射 AAV9-FOXG1 挽救了 FOXG1 综合征小鼠模型中的胼胝体缺失和其他脑部缺陷
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-06-01 DOI: 10.1016/j.omtm.2024.101275
Shin Jeon, Jaein Park, S. Likhite, Ji Hwan Moon, Dongjun Shin, Liwen Li, Kathrin C. Meyer, Jae Lee, Soo-Kyung Lee
{"title":"The postnatal injection of AAV9-FOXG1 rescues corpus callosum agenesis and other brain deficits in the mouse model of FOXG1 syndrome","authors":"Shin Jeon, Jaein Park, S. Likhite, Ji Hwan Moon, Dongjun Shin, Liwen Li, Kathrin C. Meyer, Jae Lee, Soo-Kyung Lee","doi":"10.1016/j.omtm.2024.101275","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101275","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"2 4","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141410784","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
Identifying MAGE-A4–positive tumors for TCR T-cell therapies in HLA-A*02–eligible patients 识别 MAGE-A4 阳性肿瘤，为符合 HLA-A*02 条件的患者提供 TCR T 细胞疗法
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-05-01 DOI: 10.1016/j.omtm.2024.101265
Tianjiao Wang, J. Navenot, Stavros Rafail, Cynthia Kurtis, Mark Carroll, Marian Van kerckhoven, Sofie Van Rossom, Kelly Schats, Konstantinos Avraam, R. Broad, Karen Howe, Ashley Liddle, Amber Clayton, Ruoxi Wang, Laura Quinn, Joseph P. Sanderson, Cheryl McAlpine, C. Carozza, Eric Pimpinella, Susan Hsu, F. Brophy, E. Elefant, Paige Bayer, Dennis Williams, Marcus O. Butler, Jeffrey M. Clarke, Justin Gainor, Ramaswamy Govindan, Victor Moreno, Melissa Johnson, Janet Tu, David S. Hong, G. Blumenschein
{"title":"Identifying MAGE-A4–positive tumors for TCR T-cell therapies in HLA-A*02–eligible patients","authors":"Tianjiao Wang, J. Navenot, Stavros Rafail, Cynthia Kurtis, Mark Carroll, Marian Van kerckhoven, Sofie Van Rossom, Kelly Schats, Konstantinos Avraam, R. Broad, Karen Howe, Ashley Liddle, Amber Clayton, Ruoxi Wang, Laura Quinn, Joseph P. Sanderson, Cheryl McAlpine, C. Carozza, Eric Pimpinella, Susan Hsu, F. Brophy, E. Elefant, Paige Bayer, Dennis Williams, Marcus O. Butler, Jeffrey M. Clarke, Justin Gainor, Ramaswamy Govindan, Victor Moreno, Melissa Johnson, Janet Tu, David S. Hong, G. Blumenschein","doi":"10.1016/j.omtm.2024.101265","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101265","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"1 13","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141042464","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
Prenatal AAV9-GFP administration in fetal lambs results in transduction of female germ cells and maternal exposure to virus 对胎儿羔羊进行产前 AAV9-GFP 给药会导致雌性生殖细胞转导和母体接触病毒
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-05-01 DOI: 10.1016/j.omtm.2024.101263
Beltran Borges, Antonia Varthaliti, Marisa Schwab, Maria T. Clarke, Christopher Pivetti, Nalin Gupta, C. Cadwell, G. Guibinga, Shirley Phillips, Tony Del Rio, Fatih Ozsolak, Denise Imai-Leonard, Lingling Kong, Diana J. Laird, Akos Herzeg, Charlotte J. Sumner, T. Mackenzie
{"title":"Prenatal AAV9-GFP administration in fetal lambs results in transduction of female germ cells and maternal exposure to virus","authors":"Beltran Borges, Antonia Varthaliti, Marisa Schwab, Maria T. Clarke, Christopher Pivetti, Nalin Gupta, C. Cadwell, G. Guibinga, Shirley Phillips, Tony Del Rio, Fatih Ozsolak, Denise Imai-Leonard, Lingling Kong, Diana J. Laird, Akos Herzeg, Charlotte J. Sumner, T. Mackenzie","doi":"10.1016/j.omtm.2024.101263","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101263","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"38 4","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141050626","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 blood-brain-barrier penetrant AAV gene therapy improves neurologic function in symptomatic mucolipidosis IV mice. 血脑屏障穿透型 AAV 基因疗法可改善症状性粘脂病 IV 小鼠的神经功能。
Molecular Therapy - Methods & Clinical Development Pub Date : 2024-05-01 DOI: 10.1016/j.omtm.2024.101269
Madison Sangster, Martha M. Bishop, Yizheng Yao, Jessica F. Feitor, S. Shahriar, Maxwell E. Miller, Anil K. Chekuri, Bogdan Budnik, Fengfeng Bei, Yulia Grishchuk
{"title":"A blood-brain-barrier penetrant AAV gene therapy improves neurologic function in symptomatic mucolipidosis IV mice.","authors":"Madison Sangster, Martha M. Bishop, Yizheng Yao, Jessica F. Feitor, S. Shahriar, Maxwell E. Miller, Anil K. Chekuri, Bogdan Budnik, Fengfeng Bei, Yulia Grishchuk","doi":"10.1016/j.omtm.2024.101269","DOIUrl":"https://doi.org/10.1016/j.omtm.2024.101269","url":null,"abstract":"","PeriodicalId":507212,"journal":{"name":"Molecular Therapy - Methods & Clinical Development","volume":"59 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141135290","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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