Modification of the VP1u region boosts transduction of adeno-associated virus vectors for ocular gene therapy

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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
+22 more Suk Namkung, Tapan Sharma, Thomas B. Leland, Fang Zhang, Mitchell Yip, Brian P. Rossmiller, Thomas Xue, Xiupeng Chen, Wassamon Boonying, Anoushka Lotun, Dan Wang, Qin Su, Jun Xie, Yuquan Wei, Ann Marshak‐Rothstein, Meredith S. Gregory-Ksander, András M. Komáromy, Bo Tian, Haijiang Lin, Claudio Punzo, Phillip W.L. Tai, Guangping Gao
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References: 60

Fields of Study & Subjects

Virus-based gene therapy research Retinal Development and Disorders RNA Interference and Gene Delivery

Publication details

Journal / source
Molecular Therapy
Volume
34
Issue
5
Pages
2781–2800
Publication date
30 January 2026
Language
EN