Transcriptome Profiling of Neovascularized Corneas Reveals miR-204 as a Multi-target Biotherapy Deliverable by rAAVs

by Yi Lu, Phillip W.L. Tai, Jianzhong Ai, Dominic J. Gessler, Qin Su, Xieyi Yao, Qiang Zheng, Phillip D. Zamore, Xun Xu, Guangping Gao
Published 2018-03-01

High-efficiency transduction of primary human hematopoietic stem cells and erythroid lineage-restricted expression by optimized AAV6 serotype vectors in vitro and in a murine xenog...

by Liujiang Song, Xiaomiao Li, Giridhara R Jayandharan, Yuan Wang, George V Aslanidi, Chen Ling, Li Zhong, Guangping Gao, Mervin C Yoder, Changquan Ling, Mengqun Tan, Arun Srivastava
Published 2013-01-01

Novel Roles of GATA4/6 in the Postnatal Heart Identified through Temporally Controlled, Cardiomyocyte-Specific Gene Inactivation by Adeno-Associated Virus Delivery of Cre Recombina...

by Terence W Prendiville, Haidong Guo, Zhiqiang Lin, Pingzhu Zhou, Sean M Stevens, Aibin He, Nathan VanDusen, Jinghai Chen, Li Zhong, Da-Zhi Wang, Guangping Gao, William T Pu
Published 2015-01-01

Efficacious, safe, and stable inhibition of corneal neovascularization by AAV-vectored anti-VEGF therapeutics

by Wenqi Su, Shuo Sun, Bo Tian, Phillip W.L. Tai, Yongwen Luo, Jihye Ko, Wei Zhan, Xiao Ke, Qiang Zheng, Xiaorong Li, Hua Yan, Guangping Gao, Haijiang Lin
Published 2021-09-01

Bone-Targeting AAV-Mediated Gene Silencing in Osteoclasts for Osteoporosis Therapy

by Yeon-Suk Yang, Jun Xie, Sachin Chaugule, Dan Wang, Jung-Min Kim, JiHea Kim, Phillip W.L. Tai, Seok-kyo Seo, Ellen Gravallese, Guangping Gao, Jae-Hyuck Shim
Published 2020-06-01

Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice

by Pengpeng Liu, Shun-Qing Liang, Chunwei Zheng, Esther Mintzer, Yan G. Zhao, Karthikeyan Ponnienselvan, Aamir Mir, Erik J. Sontheimer, Guangping Gao, Terence R. Flotte, Scot A. Wolfe, Wen Xue
Published 2021-04-01

Regulation of RIPK1 activation by TAK1-mediated phosphorylation dictates apoptosis and necroptosis

by Jiefei Geng, Yasushi Ito, Linyu Shi, Palak Amin, Jiachen Chu, Amanda Tomie Ouchida, Adnan Kasim Mookhtiar, Heng Zhao, Daichao Xu, Bing Shan, Ayaz Najafov, Guangping Gao, Shizuo Akira, Junying Yuan
Published 2017-08-01

AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity

by Ngoc Tam Tran, Cheryl Heiner, Kristina Weber, Michael Weiand, Daniella Wilmot, Jun Xie, Dan Wang, Alexander Brown, Sangeetha Manokaran, Qin Su, Maria L. Zapp, Guangping Gao, Phillip W.L. Tai
Published 2020-09-01

Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer

by Dan Wang, Li Zhong, Mengxin Li, Jia Li, Karen Tran, Lingzhi Ren, Ran He, Jun Xie, Richard P. Moser, Cara Fraser, Tim Kuchel, Miguel Sena-Esteves, Terence R. Flotte, Neil Aronin, Guangping Gao
Published 2018-12-01

Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette

by Sandeep R.P. Kumar, Jun Xie, Shilang Hu, Jihye Ko, Qifeng Huang, Harrison C. Brown, Alok Srivastava, David M. Markusic, Christopher B. Doering, H. Trent Spencer, Arun Srivastava, Guangping Gao, Roland W. Herzog
Published 2021-12-01

Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties

by Hung-Lun Hsu, Alexander Brown, Anna B. Loveland, Anoushka Lotun, Meiyu Xu, Li Luo, Guangchao Xu, Jia Li, Lingzhi Ren, Qin Su, Dominic J. Gessler, Yuquan Wei, Phillip W. L. Tai, Andrei A. Korostelev, Guangping Gao
Published 2020-06-01

A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates

by Dan Wang, Shaoyong Li, Dominic J. Gessler, Jun Xie, Li Zhong, Jia Li, Karen Tran, Kim Van Vliet, Lingzhi Ren, Qin Su, Ran He, Jason E. Goetzmann, Terence R. Flotte, Mavis Agbandje-McKenna, Guangping Gao
Published 2018-06-01

Start codon disruption with CRISPR/Cas9 prevents murine Fuchs’ endothelial corneal dystrophy

by Hironori Uehara, Xiaohui Zhang, Felipe Pereira, Siddharth Narendran, Susie Choi, Sai Bhuvanagiri, Jinlu Liu, Sangeetha Ravi Kumar, Austin Bohner, Lara Carroll, Bonnie Archer, Yue Zhang, Wei Liu, Guangping Gao, Jayakrishna Ambati, Albert S Jun, Balamurali K Ambati
Published 2021-06-01

Single-cell sequencing of neonatal uterus reveals an Misr2+ endometrial progenitor indispensable for fertility

by Hatice Duygu Saatcioglu, Motohiro Kano, Heiko Horn, Lihua Zhang, Wesley Samore, Nicholas Nagykery, Marie-Charlotte Meinsohn, Minsuk Hyun, Rana Suliman, Joy Poulo, Jennifer Hsu, Caitlin Sacha, Dan Wang, Guangping Gao, Kasper Lage, Esther Oliva, Mary E Morris Sabatini, Patricia K Donahoe, David Pépin
Published 2019-06-01

MARCH1 regulates insulin sensitivity by controlling cell surface insulin receptor levels

by Arvindhan Nagarajan, Max C. Petersen, Ali R. Nasiri, Gina Butrico, Annie Fung, Hai-Bin Ruan, Romy Kursawe, Sonia Caprio, Jacques Thibodeau, Marie-Claude Bourgeois-Daigneault, Lisha Sun, Guangping Gao, Sanjay Bhanot, Michael J. Jurczak, Michael R. Green, Gerald I. Shulman, Narendra Wajapeyee
Published 2016-08-01

Brain microvasculature defects and Glut1 deficiency syndrome averted by early repletion of the glucose transporter-1 protein

by Maoxue Tang, Guangping Gao, Carlos B. Rueda, Hang Yu, David N. Thibodeaux, Tomoyuki Awano, Kristin M. Engelstad, Maria-Jose Sanchez-Quintero, Hong Yang, Fanghua Li, Huapeng Li, Qin Su, Kara E. Shetler, Lynne Jones, Ryan Seo, Jonathan McConathy, Elizabeth M. Hillman, Jeffrey L. Noebels, Darryl C. De Vivo, Umrao R. Monani
Published 2017-01-01

Inhibition of miR-378a-3p by Inflammation Enhances IL-33 Levels: A Novel Mechanism of Alarmin Modulation in Ulcerative Colitis

by Karen Dubois-Camacho, David Diaz-Jimenez, David Diaz-Jimenez, Marjorie De la Fuente, Marjorie De la Fuente, Rodrigo Quera, Daniela Simian, Maripaz Martínez, Glauben Landskron, Mauricio Olivares-Morales, John A. Cidlowski, Xiaojiang Xu, Guangping Gao, Jun Xie, Jonás Chnaiderman, Ricardo Soto-Rifo, María-Julieta González, Andrea Calixto, Andrea Calixto, Marcela A. Hermoso
Published 2019-11-01

Curing hemophilia A by NHEJ-mediated ectopic F8 insertion in the mouse

by Jian-Ping Zhang, Xin-Xin Cheng, Mei Zhao, Guo-Hua Li, Jing Xu, Feng Zhang, Meng-Di Yin, Fei-Ying Meng, Xin-Yue Dai, Ya-Wen Fu, Zhi-Xue Yang, Cameron Arakaki, Ruijun Jeanna Su, Wei Wen, Wen-Tian Wang, Wanqiu Chen, Hannah Choi, Charles Wang, Guangping Gao, Lei Zhang, Tao Cheng, Xiao-Bing Zhang
Published 2019-12-01

Photoreceptor avascular privilege is shielded by soluble VEGF receptor-1

by Ling Luo, Hironori Uehara, Xiaohui Zhang, Subrata K Das, Thomas Olsen, Derick Holt, Jacquelyn M Simonis, Kyle Jackman, Nirbhai Singh, Tadashi R Miya, Wei Huang, Faisal Ahmed, Ana Bastos-Carvalho, Yun Zheng Le, Christina Mamalis, Vince A Chiodo, William W Hauswirth, Judit Baffi, Pedro M Lacal, Angela Orecchia, Napoleone Ferrara, Guangping Gao, Kim Young-hee, Yingbin Fu, Leah Owen, Romulo Albuquerque, Wolfgang Baehr, Kirk Thomas, Dean Y Li, Kakarla V Chalam, Masabumi Shibuya, Salvatore Grisanti, David J Wilson, Jayakrishna Ambati, Balamurali K Ambati
Published 2013-06-01