Pub Date : 2024-02-01DOI: 10.1016/j.omtn.2024.102148
Hali Sai, Bethany Ollington, Farah O. Rezek, Niuzheng Chai, Amelia Lane, Tassos Georgiadis, James Bainbridge, Michel Michaelides, Almudena Sacristán-Reviriego, P. Perdigão, Amy Leung, J. van der Spuy
{"title":"Effective AAV-mediated gene replacement therapy in retinal organoids modelling AIPL1-associated LCA4","authors":"Hali Sai, Bethany Ollington, Farah O. Rezek, Niuzheng Chai, Amelia Lane, Tassos Georgiadis, James Bainbridge, Michel Michaelides, Almudena Sacristán-Reviriego, P. Perdigão, Amy Leung, J. van der Spuy","doi":"10.1016/j.omtn.2024.102148","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102148","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"23 5","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139875917","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 : 2024-02-01DOI: 10.1016/j.omtn.2024.102148
Hali Sai, Bethany Ollington, Farah O. Rezek, Niuzheng Chai, Amelia Lane, Tassos Georgiadis, James Bainbridge, Michel Michaelides, Almudena Sacristán-Reviriego, P. Perdigão, Amy Leung, J. van der Spuy
{"title":"Effective AAV-mediated gene replacement therapy in retinal organoids modelling AIPL1-associated LCA4","authors":"Hali Sai, Bethany Ollington, Farah O. Rezek, Niuzheng Chai, Amelia Lane, Tassos Georgiadis, James Bainbridge, Michel Michaelides, Almudena Sacristán-Reviriego, P. Perdigão, Amy Leung, J. van der Spuy","doi":"10.1016/j.omtn.2024.102148","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102148","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"33 4","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139815885","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 : 2024-02-01DOI: 10.1016/j.omtn.2024.102141
Klaudia Kiel, Sylwia Katarzyna Król, Agnieszka Bronisz, J. Godlewski
{"title":"MiR-128-3p – a grey eminence of the human central nervous system","authors":"Klaudia Kiel, Sylwia Katarzyna Król, Agnieszka Bronisz, J. Godlewski","doi":"10.1016/j.omtn.2024.102141","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102141","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"26 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139819608","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 : 2024-02-01DOI: 10.1016/j.omtn.2024.102145
Moritz K. Jansson, Dat Tien Nguyen, Stefan Mikkat, Carolin Warnke, Marc Benjamin Janssen, Philipp Warnke, Bernd Kreikemeyer, N. Patenge
{"title":"Synthetic mRNA delivered to human cells leads to expression of Cpl-1 bacteriophage-endolysin with activity against Streptococcus pneumoniae","authors":"Moritz K. Jansson, Dat Tien Nguyen, Stefan Mikkat, Carolin Warnke, Marc Benjamin Janssen, Philipp Warnke, Bernd Kreikemeyer, N. Patenge","doi":"10.1016/j.omtn.2024.102145","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102145","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"238 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139821392","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 : 2024-02-01DOI: 10.1016/j.omtn.2024.102140
A. Cossu, Federica Melisi, Teresa Maria Rosaria Noviello, Lucia Stefania Pasquale, P. Grisolia, Carla Reale, Marco Bocchetti, Michela Falco, Chiara Tammaro, Nunzio Accardo, Francesco Longo, Salvatore Allosso, Massimo Mesolella, Raffaele Addeo, Francesco Perri, A. Ottaiano, Filippo Ricciardiello, Evžen Amler, C. Ambrosino, G. Misso, Michele Ceccarelli, M. Caraglia, M. Scrima
{"title":"MiR-449a ANTAGONIZES EMT THROUGH IL-6-MEDIATED TRANS- SIGNALING IN LARYNGEAL SQUAMOUS CANCER","authors":"A. Cossu, Federica Melisi, Teresa Maria Rosaria Noviello, Lucia Stefania Pasquale, P. Grisolia, Carla Reale, Marco Bocchetti, Michela Falco, Chiara Tammaro, Nunzio Accardo, Francesco Longo, Salvatore Allosso, Massimo Mesolella, Raffaele Addeo, Francesco Perri, A. Ottaiano, Filippo Ricciardiello, Evžen Amler, C. Ambrosino, G. Misso, Michele Ceccarelli, M. Caraglia, M. Scrima","doi":"10.1016/j.omtn.2024.102140","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102140","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"309 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139877223","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 : 2024-02-01DOI: 10.1016/j.omtn.2024.102141
Klaudia Kiel, Sylwia Katarzyna Król, Agnieszka Bronisz, J. Godlewski
{"title":"MiR-128-3p – a grey eminence of the human central nervous system","authors":"Klaudia Kiel, Sylwia Katarzyna Król, Agnieszka Bronisz, J. Godlewski","doi":"10.1016/j.omtn.2024.102141","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102141","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"22 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139879668","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 : 2024-01-01DOI: 10.1016/j.omtn.2024.102114
Yan Zhao, Kai Wang, Chun Zhao, Ning Liu, Zhihong Wang, Wenting Yang, Zewei Cheng, Luyu Zhou, Kun Wang
{"title":"The function of tRNA-derived small RNAs in cardiovascular diseases","authors":"Yan Zhao, Kai Wang, Chun Zhao, Ning Liu, Zhihong Wang, Wenting Yang, Zewei Cheng, Luyu Zhou, Kun Wang","doi":"10.1016/j.omtn.2024.102114","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102114","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"4 6","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139457009","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 : 2024-01-01DOI: 10.1016/j.omtn.2024.102122
Luís Moreira, N. Guimarães, Rita S. Santos, Joana A. Loureiro, Maria C. Pereira, Nuno F. Azevedo
{"title":"Promising strategies employing nucleic acids as antimicrobial drugs","authors":"Luís Moreira, N. Guimarães, Rita S. Santos, Joana A. Loureiro, Maria C. Pereira, Nuno F. Azevedo","doi":"10.1016/j.omtn.2024.102122","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102122","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"88 7","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139635481","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 : 2024-01-01DOI: 10.1016/j.omtn.2024.102117
Qi Tang, Katherine Y. Gross, H. Fakih, Samuel O. Jackson, Mohammad Zain Ui Abideen, Kathryn R. Monopoli, Carine Blanchard, Claire Bouix-Peter, Thibaud Portal, John E. Harris, Anastasia Khvorova, J. Alterman
{"title":"Multispecies-targeting siRNAs for the modulation of JAK1 in the skin","authors":"Qi Tang, Katherine Y. Gross, H. Fakih, Samuel O. Jackson, Mohammad Zain Ui Abideen, Kathryn R. Monopoli, Carine Blanchard, Claire Bouix-Peter, Thibaud Portal, John E. Harris, Anastasia Khvorova, J. Alterman","doi":"10.1016/j.omtn.2024.102117","DOIUrl":"https://doi.org/10.1016/j.omtn.2024.102117","url":null,"abstract":"","PeriodicalId":503104,"journal":{"name":"Molecular Therapy - Nucleic Acids","volume":"50 14","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139456355","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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