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个人简介
Research in the Gao Lab primarily involves the discovery, development and use of adeno-associated virus vectors for gene therapy of genetic diseases and the study of miRNA functions in mammals. The lab works on isolation, characterization and vectorology of novel AAV vectors from primate tissues, molecular mechanisms of AAV evolution and diversity, and molecular interactions between endogenous AAV, AAV vector, host genomes and innate RNAi defense pathways. We also develop novel strategies for rAAV gene therapy of an inherited neurodegenerative disease, Canavan Disease, using novel AAVs that can cross the blood-brain-barrier for efficient CNS gene delivery and endogenous miRNA-mediated posttranscriptional de-targeting. Another area of research interest in the lab is to explore AAV vectors for delivery of denovo synthesized biological miRNA antagonists or over expression of microRNAs to elucidate micro RNA functions in adult mammals.
研究兴趣
论文共 671 篇作者统计合作学者相似作者
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Qing Xie,Xiupeng Chen, Hong Ma, Yunxiang Zhu, Yijie Ma, Leila Jalinous, Gerald F Cox, Fiona Weaver, Jun Yang,Zachary Kennedy,Alisha Gruntman, Ailing Du,
Nathan Bamidele,Han Zhang,Xiaolong Dong,Haoyang Cheng, Nicholas Gaston, Hailey Feinzig, Hanbing Cao,Karen Kelly,Jonathan K. Watts,Jun Xie,Guangping Gao,Erik J. Sontheimer
Hao Liu,Yue Zhang, Mitchell Yip,Lingzhi Ren,Jialing Liang,Xiupeng Chen, Nan Liu, Ailing Du,Jiaming Wang,Hao Chang, Hyejin Oh,Chen Zhou,
Molecular Therapy - Methods & Clinical Developmentno. 2 (2024): 101230-101230
Shun-Qing Liang, Andrew Navia, Michelle Ramseier, Xuntao Zhou,Michele L. Martinez,Charles C. Lee,Chen Zhou, Joae Wu,Jun Xie,Qin Su, Dan Wang,Terence R Flotte,
Human Gene Therapy (2024)
Human gene therapyno. 9-10 (2024): 329-341
Shun-Qing Liang, Andrew Navia, Michelle Ramseier, Xuntao Zhou,Michele L Martinez,Charles C Lee,Chen Zhou, Joae Wu,Jun Xie,Qin Su, Dan Wang,Terence R Flotte,
Human gene therapy (2024)
JCI insight (2024)
NUCLEIC ACIDS RESEARCHno. 2 (2024): 977-997
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