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Gene therapy

Comparison of neonatal systemic and intracerebroventricular AAV9 gene therapy delivery demonstrating improved behavioral and phenotypic outcomes in a mouse model of Niemann-Pick disease, type C1

Benjamin E. Epstein, Gabrielle M. Soden, Arturo A. Incao, Avani Mylvara, Jonathan Flynn, Fatih Ozsolak, William J. Pavan

published 11 Mar 2026

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Assessing the biopotency of the rAAV9 vector In Vitro

Pratikshya Adhikari, Peter G. Nichols, Stephen M. Vorobiov, Tierra A. Bobo, Haiyan Fu

published 26 Feb 2026

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Adeno-associated vector corneal gene therapy reverses corneal clouding in a feline model of mucopolysaccharidosis VI

Brian C. Gilger, Liujiang Song, Tomoko Hasegawa, Jacklyn H. Salmon, Jacquelyn J. Bower, Naveen Vridhachalam, Elisabeth Collins, Darby Roberts, Jessica Bagel, Caitlyn Molony, Charles Vite, Matthew L. Hirsch

published 05 Dec 2025

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Synergistic enhancement of AAV gene delivery in 2D cells and 3D organoids using polybrene and hydroxychloroquine

Hyeon-Jin Na, Yongbo Shin, Seung-Hyun Kim, Seung Pil Jang, Myung Jin Son, Yong Min Choi, Hyeon Gyeol Jeon, Ok-Seon Kwon, Kyung-Sook Chung

published 14 Nov 2025

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Toward an all-in-one recombinant adeno-associated virus vector for functionally ablating the prion gene using CRISPR-Cas technology

Claire Verkuyl, Ari Belotserkovsky, Thomas Zerbes, Declan Williams, Medha R. Krishnan, Sabrina Zhu, Sophie Grunnesjӧ, Shehab Eid, Cunjie Zhang, Wenda Zhao, Leo Xu, Eleanore Lin, Teaghan O’Shea, Benjamin Draper, Andreas Jungman, Patrick Most, Gerold Schmitt-Ulms

published 07 Nov 2025

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Enrollment patterns among medicaid beneficiaries with sickle cell disease: Multistate findings from the sickle cell data collection program

Ashima Singh, Mahua Dasgupta, Hannah K. Peng, Mei Zhou, Catie Clyde, Brandon K. Attell, Jhaqueline Valle, Sarah L. Reeves, Jeffrey Huebner, Angela B. Snyder

published 27 Oct 2025

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Volumetric MRI of dorsal root ganglia as a biomarker for disease progression and response to AAV treatment in a mouse model of Fabry disease

Fuqiang Zhao, Shipeng Yuan, Charalambos Kaittanis, Mugdha Deshpande, Abirami Kugadas, Katayoun Derakhchan, Wanida Ruangsiriluk, Rizwana Islam, Natalia Boukharov, Paul McQuade, Johannes Tauscher, Christopher T. Winkelmann, Talakad G. Lohith

published 24 Oct 2025

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HMGB1-BoxA gene therapy in reversing cisplatin resistance in non-small cell lung cancer

Nattapong Puthdee, Van-Hieu Mai, Sirapat Settayanon, Pithi Chanvorachote, Chatchawit Aporntewan, Apiwat Mutirangura, Chanida Vinayanuwattikun

published 25 Jun 2025

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Gene editing therapy as a therapeutic approach for cardiovascular diseases in animal models: A scoping review

Quan Duy Vo

published 04 Jun 2025

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Intracerebroventricular administration of a modified hexosaminidase ameliorates late-stage neurodegeneration in a GM2 mouse model

Manuel E. Lopez, Daniel Wendt, Roger Lawrence, Kerui Gong, Hoonsan Ong, Bryan Yip, Joseph Chen, Linley Mangini, Britta Handyside, Alexander Giaramita, Aashish Lamichhane, Melanie Lo, Vishal Agrawal, Jeremy Van Vleet, Amanda Abolhesn, Jessica B. Felix, Isaac Villalpando, Vikas Bhat, Rolando De Angelis, Yuanbin Ru, Ayesha Khan, Sylvia Fong, Terri Christianson, Sherry Bullens, Brett E. Crawford, Stuart Bunting, Mika Aoyagi-Scharber

published 03 Jan 2025

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Tagitinin A regulates an F-box gene, CPR30, to resist tomato spotted wilt orthotospovirus (TSWV) infection in Nicotiana benthamiana

Jing Li, Xiaoman Ai, Suhua Zhang, Xue Zheng, Lizhen Zhang, Jie Zhang, Lihua Zhao

published 10 Dec 2024

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Banana fruit (Musa sp.) DNA-magnetite nanoparticles: Synthesis, characterization, and biocompatibility assays on normal and cancerous cells

David Arregui-Almeida, Martín Coronel, Karina Analuisa, Carlos Bastidas-Caldes, Santiago Guerrero, Marbel Torres, Andrea Aluisa, Alexis Debut, Werner Brämer-Escamilla, Fernanda Pilaquinga

published 14 Oct 2024

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Dynamic BMP gene expression regulation in chick RPE during recovery from short term optical defocus and form-deprivation

Yan Zhang, Qiurong Zhu, Wulian Song, Grace May Chuang, Daniel Sun, Kiana Cheung, Andreana Chou, Andrea He, Elham Shoghi, Christine F. Wildsoet

published 11 Oct 2024

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