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Molecular Therapy - Nucleic Acids最新文献

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Targeted knock-in of a NCF1 coding sequence into the endogenous NCF2 locus leads to myeloid phenotypic correction of p47 -deficient chronic granulomatous disease 将NCF1编码序列靶向敲入内源性NCF2基因座可使p47缺陷型慢性肉芽肿病的髓样表型得到纠正
Molecular Therapy - Nucleic Acids Pub Date : 2024-05-01 DOI: 10.1016/j.omtn.2024.102229
Kah Mun Siow, Merve Güngör, Dominik Wrona, Federica Raimondi, Oleksandr Pastukhov, Panagiotis Tsapogas, Timon Menzi, Michael Schmitz, Péter István Kulcsár, Gerald Schwank, Ansgar Schulz, Martin Jinek, Ute Modlich, Ulrich Siler, Janine Reichenbach
{"title":"Targeted knock-in of a NCF1 coding sequence into the endogenous NCF2 locus leads to myeloid phenotypic correction of p47 -deficient chronic granulomatous disease","authors":"Kah Mun Siow, Merve Güngör, Dominik Wrona, Federica Raimondi, Oleksandr Pastukhov, Panagiotis Tsapogas, Timon Menzi, Michael Schmitz, Péter István Kulcsár, Gerald Schwank, Ansgar Schulz, Martin Jinek, Ute Modlich, Ulrich Siler, Janine Reichenbach","doi":"10.1016/j.omtn.2024.102229","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102229","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":" 34","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141132433","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
Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells 碱基编辑可挽救小儿庞贝病患者衍生细胞中的酸α-葡萄糖苷酶功能
Molecular Therapy - Nucleic Acids Pub Date : 2024-05-01 DOI: 10.1016/j.omtn.2024.102220
Chloe Christensen, Shih-hsin Kan, Perla Andrade-Heckman, Allisandra K. Rha, Jerry F. Harb, Raymond Y. Wang
{"title":"Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells","authors":"Chloe Christensen, Shih-hsin Kan, Perla Andrade-Heckman, Allisandra K. Rha, Jerry F. Harb, Raymond Y. Wang","doi":"10.1016/j.omtn.2024.102220","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102220","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"142 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141133711","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
Engineering Miniature CRISPR-Cas Un1Cas12f1 for Efficient Base Editing 用于高效碱基编辑的微型 CRISPR-Cas Un1Cas12f1 工程技术
Molecular Therapy - Nucleic Acids Pub Date : 2024-04-01 DOI: 10.1016/j.omtn.2024.102201
Yueer Hu, Linxiao Han, Qiqin Mo, Zengming Du, Wei Jiang, Xia Wu, Jing Zheng, Xiao Xiao, Yadong Sun, Hanhui Ma
{"title":"Engineering Miniature CRISPR-Cas Un1Cas12f1 for Efficient Base Editing","authors":"Yueer Hu, Linxiao Han, Qiqin Mo, Zengming Du, Wei Jiang, Xia Wu, Jing Zheng, Xiao Xiao, Yadong Sun, Hanhui Ma","doi":"10.1016/j.omtn.2024.102201","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102201","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"695 7","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140782248","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
RNA Therapeutics in Targeting G Protein-Coupled Receptors: Recent Advances and Challenges 靶向 G 蛋白偶联受体的 RNA 疗法：最新进展与挑战
Molecular Therapy - Nucleic Acids Pub Date : 2024-04-01 DOI: 10.1016/j.omtn.2024.102195
Wanjun Yuan, Xiangyang Shi, Leo Tsz On Lee
{"title":"RNA Therapeutics in Targeting G Protein-Coupled Receptors: Recent Advances and Challenges","authors":"Wanjun Yuan, Xiangyang Shi, Leo Tsz On Lee","doi":"10.1016/j.omtn.2024.102195","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102195","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"87 11","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140758754","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
N4-acetylcytidine acetylation of neurexin 2 in the spinal dorsal horn regulates hypersensitivity in a rat model of cancer-induced bone pain 脊髓背角神经肽 2 的 N4-乙酰胞嘧啶乙酰化调节癌症诱发骨痛大鼠模型的超敏反应
Molecular Therapy - Nucleic Acids Pub Date : 2024-04-01 DOI: 10.1016/j.omtn.2024.102200
Longsheng Xu, Shang Zheng, Liping Chen, Lei Yang, Shuyao Zhang, Beibei Liu, Kangli Shen, Qinli Feng, Qinghe Zhou, Ming Yao
{"title":"N4-acetylcytidine acetylation of neurexin 2 in the spinal dorsal horn regulates hypersensitivity in a rat model of cancer-induced bone pain","authors":"Longsheng Xu, Shang Zheng, Liping Chen, Lei Yang, Shuyao Zhang, Beibei Liu, Kangli Shen, Qinli Feng, Qinghe Zhou, Ming Yao","doi":"10.1016/j.omtn.2024.102200","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102200","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"49 4","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140791774","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
ac4C-AFL: A high-precision identification of human mRNA N4-acetylcytidine sites based on adaptive feature representation learning ac4C-AFL：基于自适应特征表征学习的人类 mRNA N4-乙酰胞嘧啶位点高精度识别技术
Molecular Therapy - Nucleic Acids Pub Date : 2024-04-01 DOI: 10.1016/j.omtn.2024.102192
Nhat Truong Pham, Annie Terrina Terrance, Young-Jun Jeon, Rajan Rakkiyapan, Balachandran Manavalan
{"title":"ac4C-AFL: A high-precision identification of human mRNA N4-acetylcytidine sites based on adaptive feature representation learning","authors":"Nhat Truong Pham, Annie Terrina Terrance, Young-Jun Jeon, Rajan Rakkiyapan, Balachandran Manavalan","doi":"10.1016/j.omtn.2024.102192","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102192","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"69 6","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140759079","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
Selective targeting of chemically modified miR-34a to prostate cancer using a small molecule ligand and an endosomal escape agent 利用小分子配体和内体逃逸剂选择性地将化学修饰的 miR-34a 靶向前列腺癌
Molecular Therapy - Nucleic Acids Pub Date : 2024-04-01 DOI: 10.1016/j.omtn.2024.102193
Ahmed M. Abdelaal, I. Sohal, Shreyas Iyer, Kasireddy Sudarshan, Esteban A. Orellana, Kenan E. Ozcan, Andrea P. dos Santos, Philip S. Low, A. Kasinski
{"title":"Selective targeting of chemically modified miR-34a to prostate cancer using a small molecule ligand and an endosomal escape agent","authors":"Ahmed M. Abdelaal, I. Sohal, Shreyas Iyer, Kasireddy Sudarshan, Esteban A. Orellana, Kenan E. Ozcan, Andrea P. dos Santos, Philip S. Low, A. Kasinski","doi":"10.1016/j.omtn.2024.102193","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102193","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"73 41","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140794589","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
Expanding RNA editing toolkit using an IDR-based strategy 利用基于 IDR 的策略扩展 RNA 编辑工具包
Molecular Therapy - Nucleic Acids Pub Date : 2024-04-01 DOI: 10.1016/j.omtn.2024.102190
Minghui Di, Junjun Lv, Zhengyu Jing, Yijie Yang, Kunlun Yan, Jianguo Wu, Jianyang Ge, Simone Rauch, Bryan Dickinson, Tian Chi
{"title":"Expanding RNA editing toolkit using an IDR-based strategy","authors":"Minghui Di, Junjun Lv, Zhengyu Jing, Yijie Yang, Kunlun Yan, Jianguo Wu, Jianyang Ge, Simone Rauch, Bryan Dickinson, Tian Chi","doi":"10.1016/j.omtn.2024.102190","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102190","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"49 8","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140759216","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
Enhanced transgene expression from single-stranded AAV vectors in human cells in vitro and in murine hepatocytes in vivo 增强单链 AAV 载体在体外人体细胞和体内小鼠肝细胞中的转基因表达
Molecular Therapy - Nucleic Acids Pub Date : 2024-04-01 DOI: 10.1016/j.omtn.2024.102196
Yuan Lu, Chen Ling, Jakob Shoti, Hua Yang, Aneesha Nath, Geoffrey D. Keeler, Keyun Qing, Arun Srivastava
{"title":"Enhanced transgene expression from single-stranded AAV vectors in human cells in vitro and in murine hepatocytes in vivo","authors":"Yuan Lu, Chen Ling, Jakob Shoti, Hua Yang, Aneesha Nath, Geoffrey D. Keeler, Keyun Qing, Arun Srivastava","doi":"10.1016/j.omtn.2024.102196","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102196","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"28 12","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140796887","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
CD3 aptamers promote expansion and persistence of tumor-reactive T cells for adoptive T-cell therapy in cancer CD3 嵌合体可促进肿瘤反应性 T 细胞的扩增和持久性，用于癌症的 T 细胞疗法
Molecular Therapy - Nucleic Acids Pub Date : 2024-04-01 DOI: 10.1016/j.omtn.2024.102198
A. P. Menon, H. Villanueva, Daniel Meraviglia-Crivelli, H. V. van Santen, Joschka Hellmeier, Angelina Zheleva, Francesca Nonateli, Timo Peters, T. Wachsmann, Mercedes Hernandez-Rueda, Johannes B Huppa, Gerhard J. Schütz, Eva Sevcsik, Beatriz Moreno, Fernando Pastor
{"title":"CD3 aptamers promote expansion and persistence of tumor-reactive T cells for adoptive T-cell therapy in cancer","authors":"A. P. Menon, H. Villanueva, Daniel Meraviglia-Crivelli, H. V. van Santen, Joschka Hellmeier, Angelina Zheleva, Francesca Nonateli, Timo Peters, T. Wachsmann, Mercedes Hernandez-Rueda, Johannes B Huppa, Gerhard J. Schütz, Eva Sevcsik, Beatriz Moreno, Fernando Pastor","doi":"10.1016/j.omtn.2024.102198","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102198","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"116 ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140792836","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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