Pub Date : 2024-05-01DOI: 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}
Pub Date : 2024-05-01DOI: 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}
Pub Date : 2024-04-01DOI: 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}
Pub Date : 2024-04-01DOI: 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}
Pub Date : 2024-04-01DOI: 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}
Pub Date : 2024-04-01DOI: 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}
Pub Date : 2024-04-01DOI: 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}
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