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Anti-HIV-1 HSPC-based gene therapy with safety kill switch to defend against and attack HIV-1 infectio
Author: Guo, Qi; Parikh, Keval; Zhang, Jian; Brinkley, Alexander; Chen, Grace; Jakramonpreeya, Natnicha; Zhen, Anjie; An, Dong Sung
Journal: MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT. 2025; Vol. 33, Issue 2, pp. -. DOI: 10.1016/j.omtm.2025.101486
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Self-silencing adenovirus enables precise infectious titration of recombinant adeno-associated viral vector
Author: Fustinoni, Chloe; Liu, Xiang; Chen, Zhi; Cawood, Ryan; Valenti, Weimin; Patricio, Maria I.; Su, Weiheng
Journal: MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT. 2025; Vol. 33, Issue 2, pp. -. DOI: 10.1016/j.omtm.2025.101492
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The clinical quality management system of advanced therapy medicinal products in the hospital setting: A scoping revie
Author: Shi, Junnan; Yang, Jinagya; Zheng, Yu; Wong, Phyllis Hio Hong; Hu, Hao; Ung, Carolina Oi Lam
Journal: MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT. 2025; Vol. 33, Issue 2, pp. -. DOI: 10.1016/j.omtm.2025.101485
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Preclinical studies of an AAV8-CYP4V2 gene therapy VGR-R01 for the treatment of Bietti crystalline dystroph
Author: Luo, Wenjing; Guo, Lu; Lu, Liang; Huang, Nan; Tao, Yezheng; Zhang, Yufei; Cao, Yanrong; Tian, Shin-Shay; Zhao, Xiaoping; Zhu, Xi
Journal: MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT. 2025; Vol. 33, Issue 2, pp. -. DOI: 10.1016/j.omtm.2025.101460
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Lipid nanoparticles: Composition, formulation, and applicatio
Author: Xu, Sijia; Hu, Zhenzhen; Song, Fenglin; Xu, Ying; Han, Xuexiang
Journal: MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT. 2025; Vol. 33, Issue 2, pp. -. DOI: 10.1016/j.omtm.2025.101463
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Efficacy and preliminary safety assessment of EXG001-307 AAV gene therapy for spinal muscular atroph
Author: Song, Chunjuan; Wang, Qingzeng; Zhu, Peixin; Li, Junhui; Dai, Li; Hu, Wei; Yang, Wenshu; Zhang, Runyuan; Wang, Dandan; Li, Zhongwan; Chen, Shuyuan; Wang, Lijun; Ye, Guojie; Wu, Zhenhua
Journal: MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT. 2025; Vol. 33, Issue 2, pp. -. DOI: 10.1016/j.omtm.2025.101475
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The combination of rAAV pseudo-lipid nanoparticle and triamcinolone acetonide enables multi-administration to live
Author: Gan, Chunmei; Leng, Mi; Liu, Yu; Zheng, Zhaoyue; He, Siwu; Qiao, Wen; Xiao, Lin; Xiao, Yao; Ye, Jingya; Zhou, Lixing; Zhou, Jiao; Xiao, Boduan; Zhao, Wenxin; Yang, Jiamei; Wu, Aohan; Zhang, Huiyuan; Hu, Hongbo; Cen, Xiaobo; Qian, Zhiyong; Dong, Haohao; Valencia, C. Alexander; Dai, Lunzhi; Chow, Hoi Yee; Zhang, Lei; Dong, Biao
Journal: MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT. 2025; Vol. 33, Issue 1, pp. -. DOI: 10.1016/j.omtm.2024.101399
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Intravenous esketamine in pediatric Rett syndrome: An open-label, early phase 1 pilot stud
Author: Li, Huiping; Liu, Shu; Lin, Caimei; Wu, Yuchao; Wu, Xiuping; Huang, Yukun; Wu, Yajun; Tong, Xiubin; Xu, Xiu
Journal: MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT. 2025; Vol. 33, Issue 1, pp. -. DOI: 10.1016/j.omtm.2025.101413
