Pub Date : 2026-02-03DOI: 10.1016/j.ymthe.2026.01.036
Jing Zhou, Yue Wan, Michael G. Anderson, Elia J. Duh, Jonathan B. Demb, Xinzheng Guo, Bo Chen
{"title":"CaMKII-based gene therapy protects retinal ganglion cells in a broad range of disease: ischemic retinopathy and congenital glaucoma","authors":"Jing Zhou, Yue Wan, Michael G. Anderson, Elia J. Duh, Jonathan B. Demb, Xinzheng Guo, Bo Chen","doi":"10.1016/j.ymthe.2026.01.036","DOIUrl":"https://doi.org/10.1016/j.ymthe.2026.01.036","url":null,"abstract":"","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":"9 1","pages":""},"PeriodicalIF":12.4,"publicationDate":"2026-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146109918","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 : 2026-02-03DOI: 10.1016/j.ymthe.2026.01.038
Jack M.P. Castelli, Katrina Poljakov, Youngseo Jwa, Rachel Cunningham, Molly E. Cassidy, Matthew D. Gray, Jocelyn N. Sanchez Gaytan, Mark R. Enstrom, Grady Gastelum, Zhongchen Wang, Jonathan D. Linton, Anthony Rongvaux, Justin J. Taylor, Jennifer E. Adair
{"title":"In vivo production of an anti-HIV antibody in mice by non-viral gene knock-in in primate hematopoietic stem and progenitor cells","authors":"Jack M.P. Castelli, Katrina Poljakov, Youngseo Jwa, Rachel Cunningham, Molly E. Cassidy, Matthew D. Gray, Jocelyn N. Sanchez Gaytan, Mark R. Enstrom, Grady Gastelum, Zhongchen Wang, Jonathan D. Linton, Anthony Rongvaux, Justin J. Taylor, Jennifer E. Adair","doi":"10.1016/j.ymthe.2026.01.038","DOIUrl":"https://doi.org/10.1016/j.ymthe.2026.01.038","url":null,"abstract":"","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":"1 1","pages":""},"PeriodicalIF":12.4,"publicationDate":"2026-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146109915","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 : 2026-01-30DOI: 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}
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