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A novel gene delivery platform based on a commensal human anellovirus demonstrates transduction in multiple tissue types
Author:
Cato Prince, George Bounoutas, Bolu Zhou, Waseem Raja, Isabella Gold, Riana Pozsgai, Parmi Thakker, Nicole Boisvert, Christopher Reardon, Stephanie Thurmond, Erin Ozturk, Rajendra Boggavarapu, Jenna Antonucci Johnson, Patricio Jeraldo, Simeon Springer, Lovepreet Chahal, Emily Stead, Cameron Dodier, Bryan Vought, Maciej Nogalski, Dhananjay Nawandar, Christopher Wright, Ashley Mackey, Geoffrey Parsons, Joseph Cabral
Journal: Molecular Therapy
Doi: https://www.doi.org/10.1016/j.omtm.2025.101597
2025/12/11 -
Single-nucleus RNA sequencing reveals GABAergic vulnerability and reactive gliosis driven by loss of TDP-43
Author:
Rashmi Thapa, Navin Adhikari, Sishir Gautam, Mingming Sun, Sierra McOmie, Viyaleta Davydzenka, Derek Smith, Jack Syring, Harlie Kaligis, Jade Marie Kosmicki, Rong Chen, Yun Li
Journal: iScience
Doi: https://www.doi.org/10.1016/j.isci.2025.113745
2025/11/21 -
In-Depth Optimization and Systematic Evaluation of a Recombinant AAV-Based Hepatic-Targeted Gene Knockdown Murine Model
Author:
Ying Zhu, Guohua You, Yong Jiang, Keyu Yin, Rui Wang, Changhong Li, Xiaodan Zhang, Zhiping Yan, Mingjie Ding, Wenzhi Guo, Lele Zhang
Journal: FASEB
Doi: https://www.doi.org/10.1096/fj.202502602R
2025/10/30 -
Transcription factor activator protein 4 (AP4)–mediated intrinsic control of axon regeneration
Author:
Ngan Pan Bennett Au, Yumeng Gan, Xinyu Chen, Fuying Gao, Christopher Kai-Shun Leung, and Chi Him Eddie Ma
Journal: SCIENCE ADVANCES
Doi: https://www.doi.org/10.1126/sciadv.adu3917
2025/10/29 -
Noncanonical target-strand cytosine base editing via engineered Un1Cas12f1 platform
Author:
Ziguo Song, Junfan Guo, Zhanqing Fan, Shuhong Huang, Guanglei Li, Zichang Zhao, Bingchun Chen, Shisheng Huang, Wenxin Zheng, Yinghui Wei, Yulin Chen, Xingxu Huang, Jianghuai Liu, Lina Wu & Xiaolong Wang
Journal: Nature Communications
Doi: https://www.doi.org/10.1038/s41467-025-64562-0
2025/10/28 -
AAV-mediated CSNK2B gene replacement rescues ASD-relevant phenotypes and establishes EEG biomarkers for translation in Csnk2b haploinsufficient mice
Author:
Chaodong Ding, Xiaqing Wang, Yiting Yuan, Yuefang Zhang, Yixin Hu, Ailian Du, Zilong Qiu
Journal: BioRxiv
Doi: https://www.doi.org/10.1101/2025.10.23.684260
2025/10/24 -
Chronic HBV infection impairs the glucose metabolism and effector function of NK cells via HBsAg/IL-15/mTOR axis
Author:
Yating Yu, Zixuan Wang, Ailu Yang, Yucan Wang, Cuiping Bao, Li Zhuo, Qiuju Han, Huajun Zhao & Jian Zhang
Journal: Cell Death & Disease
Doi: https://www.doi.org/10.1038/s41419-025-08069-y
2025/10/13 -
Targeted genome editing of ZKSCAN3 mitigates the neurotoxicity caused by mutant HTT (huntingtin) in a Huntington disease animal model and three-dimensional cell culture of Huntington disease
Author:
Hyun Jung Park, JiYeon Kim, Jiwoo Choi, Chongsuk Ryou, Eunji Shin & Jae Young Lee
Journal: Autophagy
Doi: https://www.doi.org/10.1080/15548627.2025.2569965
2025/10/10 -
Ototoxicity-induced c-Fos activation underlies the regenerative capacity of the vestibular sensory epithelia
Author:
Yunzhong Zhang, Dan You, Chenhao Che, Xinyuan Wang, Huawei Li, Yi-Quan Tang, Shan Sun
Journal: Cell Communication and Signaling
Doi: https://www.doi.org/10.1186/s12964-025-02446-y
2025/10/8 -
HnRNP M expression rescues neurodegeneration in neuronal intranuclear inclusion disease mouse model by restoring dysregulated RNA splicing and transcription
Author:
Yongcheng Pan, Yangping Li, Ying Jiang, Xinhui Wang, Juan Wan, Qiying Sun, Yun Tian, Lu Shen, Hong Jiang, Beisha Tang, Bing Yao, Qiong Liu
Journal: Cell and Bioscience
Doi: https://www.doi.org/10.1186/s13578-025-01477-9
2025/10/6 -
AAV-mediated allele-specific silencing alleviates neuropathology in a novel non-human primate model of Spinocerebellar ataxia type 3
Author:
Carina Henriques, Diana Duarte Lobo, Ana Carolina Silva, Ana Rita Fernandes, Miguel Monteiro Lopes, Audrey Fayard, Caroline Jan, Sophie Lecourtois, Martine Guillermier, Julien Flament, João Castelhano, Miguel Castelo-Branco, Rui Caetano Oliveira, Philippe Hantraye, Padmaja Yalamanchili, Steven de Marco, Romina Aron Badin, Luís Pereira de Almeida and Rui Jorge Nobre
Journal: BioRxiv
Doi: https://www.doi.org/10.1101/2025.10.02.680027
2025/10/2 -
rAAV-Delivered Bicistronic Artificial microRNAs for Allele-Specific Silencing Improve Motor and Molecular Outcomes in Spinocerebellar Ataxia Type 3
Author:
Ana Carolina Silva, Carina Henriques, Diana D. Lobo, Ana Rita Fernandes, Miguel M. Lopes, Kevin Leandro, Dina Pereira, Sónia P. Duarte, Sara M. Lopes, Magda M. Santana, Amal Dakka, Steve De Marco, Marla Weetall, Jana Narasimhan, Anu Bhattacharyya, Rui Jorge Nobre and Luís Pereira de Almeida
Journal: BioRxiv
Doi: https://www.doi.org/10.1101/2025.10.02.680011
2025/10/2