Molecular therapy : the journal of the American Society of Gene Therapy最新文献

{"title":"Evolution of the clinical-stage hyperactive TcBuster transposase as a platform for robust non-viral production of adoptive cellular therapies.","authors":"Joseph G. Skeate, E. Pomeroy, Nicholas J. Slipek, Bryan J Jones, B. Wick, J. Chang, Walker S. Lahr, Erin M. Stelljes, Xiaobai Patrinostro, Blake Barnes, Trevor Zarecki, Joshua B. Krueger, Jacob E Bridge, Gabrielle M. Robbins, Madeline D McCormick, John R Leerar, Kari T Wenzel, Kathlyn Hornberger, Kirsti Walker, Dalton Smedley, D. Largaespada, Neil Otto, Beau R. Webber, B. Moriarity","doi":"10.1016/j.ymthe.2024.04.024","DOIUrl":"https://doi.org/10.1016/j.ymthe.2024.04.024","url":null,"abstract":"","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":"86 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140797703","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}

{"title":"The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease.","authors":"Antonio Monaco, Veronica Maffia, Nicolina Cristina Sorrentino, Irene Sambri, Yulia Ezhova, Teresa Giuliano, Vincenzo Cacace, Edoardo Nusco, Maria De Risi, Elvira De Leonibus, Thomas Schrader, Frank-Gerrit Klärner, Gal Bitan, Alessandro Fraldi","doi":"10.1016/j.ymthe.2022.10.001","DOIUrl":"https://doi.org/10.1016/j.ymthe.2022.10.001","url":null,"abstract":"","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"3499"},"PeriodicalIF":12.4,"publicationDate":"2022-11-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9637752/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40342981","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Corneal fibrosis abrogation by a localized AAV-mediated inhibitor of differentiation 3 (Id3) gene therapy in rabbit eyes in vivo.","authors":"Suneel Gupta, Michael K Fink, Duraisamy Kempuraj, Nishant R Sinha, Lynn M Martin, Landon M Keele, Prashant R Sinha, Elizabeth A Giuliano, Nathan P Hesemann, Sudhanshu P Raikwar, Shyam S Chaurasia, Rajiv R Mohan","doi":"10.1016/j.ymthe.2022.08.015","DOIUrl":"https://doi.org/10.1016/j.ymthe.2022.08.015","url":null,"abstract":"","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"3334"},"PeriodicalIF":12.4,"publicationDate":"2022-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9552807/pdf/main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40645223","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The paradigm of immune escape by SARS-CoV-2 variants and strategies for repositioning subverted mAbs against escaped VOCs.","authors":"Masaud Shah,&nbsp;Hyun Goo Woo","doi":"10.1016/j.ymthe.2022.08.020","DOIUrl":"https://doi.org/10.1016/j.ymthe.2022.08.020","url":null,"abstract":"<p><p>The perpetual emergence of SARS-CoV-2 variants is a serious issue that makes it difficult for the therapeutic antibodies and vaccines to end the COVID-19 pandemic. This article discusses the trend of increasing host fitness and immune escape by the virus and how to devise computational strategies for antibodies design and their affinity maturation against emerging SARS-CoV-2 variants.</p>","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"3101-3105"},"PeriodicalIF":12.4,"publicationDate":"2022-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9462941/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"33458338","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"mRNA-LNP vaccines tuned for systemic immunization induce strong antitumor immunity by engaging splenic immune cells.","authors":"Sanne Bevers,&nbsp;Sander A A Kooijmans,&nbsp;Elien Van de Velde,&nbsp;Martijn J W Evers,&nbsp;Sofie Seghers,&nbsp;Jerney J J M Gitz-Francois,&nbsp;Nicky C H van Kronenburg,&nbsp;Marcel H A M Fens,&nbsp;Enrico Mastrobattista,&nbsp;Lucie Hassler,&nbsp;Helena Sork,&nbsp;Taavi Lehto,&nbsp;Kariem E Ahmed,&nbsp;Samir El Andaloussi,&nbsp;Katja Fiedler,&nbsp;Karine Breckpot,&nbsp;Michael Maes,&nbsp;Diane Van Hoorick,&nbsp;Thierry Bastogne,&nbsp;Raymond M Schiffelers,&nbsp;Stefaan De Koker","doi":"10.1016/j.ymthe.2022.07.007","DOIUrl":"https://doi.org/10.1016/j.ymthe.2022.07.007","url":null,"abstract":"<p><p>mRNA vaccines have recently proved to be highly effective against SARS-CoV-2. Key to their success is the lipid-based nanoparticle (LNP), which enables efficient mRNA expression and endows the vaccine with adjuvant properties that drive potent antibody responses. Effective cancer vaccines require long-lived, qualitative CD8 T cell responses instead of antibody responses. Systemic vaccination appears to be the most effective route, but necessitates adaptation of LNP composition to deliver mRNA to antigen-presenting cells. Using a design-of-experiments methodology, we tailored mRNA-LNP compositions to achieve high-magnitude tumor-specific CD8 T cell responses within a single round of optimization. Optimized LNP compositions resulted in enhanced mRNA uptake by multiple splenic immune cell populations. Type I interferon and phagocytes were found to be essential for the T cell response. Surprisingly, we also discovered a yet unidentified role of B cells in stimulating the vaccine-elicited CD8 T cell response. Optimized LNPs displayed a similar, spleen-centered biodistribution profile in non-human primates and did not trigger histopathological changes in liver and spleen, warranting their further assessment in clinical studies. Taken together, our study clarifies the relationship between nanoparticle composition and their T cell stimulatory capacity and provides novel insights into the underlying mechanisms of effective mRNA-LNP-based antitumor immunotherapy.</p>","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"3078-3094"},"PeriodicalIF":12.4,"publicationDate":"2022-09-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/45/10/main.PMC9273295.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40496600","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Preclinical and Clinical Demonstration of Immunogenicity by mRNA Vaccines against H10N8 and H7N9 Influenza Viruses.","authors":"Kapil Bahl,&nbsp;Joe J Senn,&nbsp;Olga Yuzhakov,&nbsp;Alex Bulychev,&nbsp;Luis A Brito,&nbsp;Kimberly J Hassett,&nbsp;Michael E Laska,&nbsp;Mike Smith,&nbsp;Örn Almarsson,&nbsp;James Thompson,&nbsp;Amilcar Mick Ribeiro,&nbsp;Mike Watson,&nbsp;Tal Zaks,&nbsp;Giuseppe Ciaramella","doi":"10.1016/j.ymthe.2022.07.013","DOIUrl":"https://doi.org/10.1016/j.ymthe.2022.07.013","url":null,"abstract":"","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"2874"},"PeriodicalIF":12.4,"publicationDate":"2022-08-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/b8/f4/main.PMC9372314.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40597528","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Base-edited cynomolgus monkeys mimic core symptoms of STXBP1 encephalopathy.","authors":"Zongyang Lu,&nbsp;Siting He,&nbsp;Jian Jiang,&nbsp;Ling Zhuang,&nbsp;Yan Wang,&nbsp;Guang Yang,&nbsp;Xiaoyu Jiang,&nbsp;Yanhong Nie,&nbsp;Jiqiang Fu,&nbsp;Xiaotong Zhang,&nbsp;Yong Lu,&nbsp;Xinyan Bian,&nbsp;Hung-Chun Chang,&nbsp;Zhiqi Xiong,&nbsp;Xingxu Huang,&nbsp;Zhen Liu,&nbsp;Qiang Sun","doi":"10.1016/j.ymthe.2022.06.020","DOIUrl":"https://doi.org/10.1016/j.ymthe.2022.06.020","url":null,"abstract":"","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"2869-2873"},"PeriodicalIF":12.4,"publicationDate":"2022-08-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/33/d2/main.PMC9372315.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40479027","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Retraction Notice to: METTL14 Suppresses CRC Progression via Regulating N6-Methyladenosine-Dependent Primary miR-375 Processing.","authors":"Xiaoxiang Chen,&nbsp;Mu Xu,&nbsp;Xueni Xu,&nbsp;Kaixuan Zeng,&nbsp;Xiangxiang Liu,&nbsp;Li Sun,&nbsp;Bei Pan,&nbsp;Bangshun He,&nbsp;Yuqin Pan,&nbsp;Huiling Sun,&nbsp;Xinyi Xia,&nbsp;Shukui Wang","doi":"10.1016/j.ymthe.2022.03.017","DOIUrl":"https://doi.org/10.1016/j.ymthe.2022.03.017","url":null,"abstract":"","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"2640"},"PeriodicalIF":12.4,"publicationDate":"2022-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/2c/fe/main.PMC9263224.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40478642","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Retraction Notice to: Comprehensive Analysis of the Expression and Prognosis for E2Fs in Human Breast Cancer.","authors":"Cheng-Cao Sun,&nbsp;Shu-Jun Li,&nbsp;Wei Hu,&nbsp;Jian Zhang,&nbsp;Qun Zhou,&nbsp;Cong Liu,&nbsp;Lin-Lin Li,&nbsp;Yi-Yan Songyang,&nbsp;Feng Zhang,&nbsp;Zhen-Long Chen,&nbsp;Guang Li,&nbsp;Zhuo-Yue Bi,&nbsp;Yong-Yi Bi,&nbsp;Feng-Yun Gong,&nbsp;Tao Bo,&nbsp;Zhan-Peng Yuan,&nbsp;Wei-Dong Hu,&nbsp;Bo-Tao Zhan,&nbsp;Qian Zhang,&nbsp;Qi-Qiang He,&nbsp;De-Jia Li","doi":"10.1016/j.ymthe.2022.03.018","DOIUrl":"https://doi.org/10.1016/j.ymthe.2022.03.018","url":null,"abstract":"","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"2639"},"PeriodicalIF":12.4,"publicationDate":"2022-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9263386/pdf/main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40478641","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Retraction Notice to: miR-135b Delivered by Gastric Tumor Exosomes Inhibits FOXO1 Expression in Endothelial Cells and Promotes Angiogenesis.","authors":"Ming Bai,&nbsp;Jialu Li,&nbsp;Haiou Yang,&nbsp;Haiyang Zhang,&nbsp;Zhengyang Zhou,&nbsp;Ting Deng,&nbsp;Kegan Zhu,&nbsp;Tao Ning,&nbsp;Qian Fan,&nbsp;Guoguang Ying,&nbsp;Yi Ba","doi":"10.1016/j.ymthe.2022.03.021","DOIUrl":"https://doi.org/10.1016/j.ymthe.2022.03.021","url":null,"abstract":"","PeriodicalId":519519,"journal":{"name":"Molecular therapy : the journal of the American Society of Gene Therapy","volume":" ","pages":"2636"},"PeriodicalIF":12.4,"publicationDate":"2022-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9263227/pdf/main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40478638","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}