Pub Date : 2026-01-30DOI: 10.1016/j.ymthe.2026.01.029
Mengtian Cui, Shun-Yun Cheng, Jialing Liang, Jackson McGowan, Meiyu Xu, Guangchao Xu, Li Luo, Joae Wu, Christine D. Harman, Amanda L. Jacobson, Lydia E. Kapeller, Melaney A. Mayes, Suk Namkung, Tapan Sharma, Thomas B. Leland, Fang Zhang, Mitchell Yip, Brian Rossmiller, Thomas Xue, Xiupeng Chen, Wassamon Boonying, Anoushka Lotun, Dan Wang, Qin Su, Jun Xie, Yuquan Wei, Ann M. Rothstein, Meredith S. Gregory-Ksander, Andras Komaromy, Bo Tian, Haijiang Lin, Claudio Punzo, Phillip W.L. Tai, Guangping Gao
{"title":"Modification of the VP1u region boosts transduction of adeno-associated virus vectors for ocular gene therapy","authors":"Mengtian Cui, Shun-Yun Cheng, Jialing Liang, Jackson McGowan, Meiyu Xu, Guangchao Xu, Li Luo, Joae Wu, Christine D. Harman, Amanda L. Jacobson, Lydia E. Kapeller, Melaney A. Mayes, Suk Namkung, Tapan Sharma, Thomas B. Leland, Fang Zhang, Mitchell Yip, Brian Rossmiller, Thomas Xue, Xiupeng Chen, Wassamon Boonying, Anoushka Lotun, Dan Wang, Qin Su, Jun Xie, Yuquan Wei, Ann M. Rothstein, Meredith S. Gregory-Ksander, Andras Komaromy, Bo Tian, Haijiang Lin, Claudio Punzo, Phillip W.L. Tai, Guangping Gao","doi":"10.1016/j.ymthe.2026.01.029","DOIUrl":"https://doi.org/10.1016/j.ymthe.2026.01.029","url":null,"abstract":"","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":"7 1","pages":""},"PeriodicalIF":12.4,"publicationDate":"2026-01-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146071577","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2026-01-29DOI: 10.1016/j.ymthe.2026.01.034
Lilit Antonyan, Linda Bossini, Xin Zhang, Anjie Ni, Marc-Daniel Hagel, Eva Baronchelli, Martina Geusa, Huashan Peng, Amelia Semenak, Peter Fleming, Shaima Alsuwaidi, Ying Zhang, Beili Hu, Diletta Fontana, Valentina Crippa, Stefania Codazzi, Julia Macintosh, Andres Arriaga-Sierra, Basil Khan, Nuwan Hettige, Malvin Jefri, Yijing Liang, Stephanie Klein, André du Toit, Nhu T.N. Phan, Andreas Schuppert, Yang Zhou, Rocco Piazza, Keith Murai, Sebastien Jacquemont, Alessandro Sessa, Carl Ernst
{"title":"Prevention of hydrocephalus with a small oligonucleotide","authors":"Lilit Antonyan, Linda Bossini, Xin Zhang, Anjie Ni, Marc-Daniel Hagel, Eva Baronchelli, Martina Geusa, Huashan Peng, Amelia Semenak, Peter Fleming, Shaima Alsuwaidi, Ying Zhang, Beili Hu, Diletta Fontana, Valentina Crippa, Stefania Codazzi, Julia Macintosh, Andres Arriaga-Sierra, Basil Khan, Nuwan Hettige, Malvin Jefri, Yijing Liang, Stephanie Klein, André du Toit, Nhu T.N. Phan, Andreas Schuppert, Yang Zhou, Rocco Piazza, Keith Murai, Sebastien Jacquemont, Alessandro Sessa, Carl Ernst","doi":"10.1016/j.ymthe.2026.01.034","DOIUrl":"https://doi.org/10.1016/j.ymthe.2026.01.034","url":null,"abstract":"","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":"28 1","pages":""},"PeriodicalIF":12.4,"publicationDate":"2026-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146072195","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2026-01-29DOI: 10.1016/j.ymthe.2026.01.033
Claire E. Tocheny, Julianna E. Buchwald, Christopher D. Dahlke, Hassan H. Fakih, Jennifer S. Morgan, Ashley Summers, Christi A. Silva, Samuel O. Jackson, Ji-Sun Lee, Michael-Anthony Card, Dimas Echeverria, Cornelia Peterson, Arthur M. Mercurio, Anastasia Khvorova, Leslie M. Shaw
{"title":"Therapeutic delivery of albumin-binding siRNA targeting IRS2 to diverse cell types reduces mammary tumor growth","authors":"Claire E. Tocheny, Julianna E. Buchwald, Christopher D. Dahlke, Hassan H. Fakih, Jennifer S. Morgan, Ashley Summers, Christi A. Silva, Samuel O. Jackson, Ji-Sun Lee, Michael-Anthony Card, Dimas Echeverria, Cornelia Peterson, Arthur M. Mercurio, Anastasia Khvorova, Leslie M. Shaw","doi":"10.1016/j.ymthe.2026.01.033","DOIUrl":"https://doi.org/10.1016/j.ymthe.2026.01.033","url":null,"abstract":"","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":"260 1","pages":""},"PeriodicalIF":12.4,"publicationDate":"2026-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146072197","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}