Size does matter: overcoming the adeno-associated virus packaging limit

por Flotte, Terence R
Publicado 2000

Liver targeting with rAAV7: balancing tropism with immune profiles

por Flotte, Terence R.
Publicado 2021

CRISPR/Cas-Dependent and Nuclease-Free In Vivo Therapeutic Gene Editing

por Dasgupta, Ishani, Flotte, Terence R., Keeler, Allison M.
Publicado 2021

Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer

por Arjomandnejad, Motahareh, Dasgupta, Ishani, Flotte, Terence R., Keeler, Allison M.
Publicado 2023

No Immune Responses by the Expression of the Yeast Ndi1 Protein in Rats

por Marella, Mathieu, Seo, Byoung Boo, Flotte, Terence R., Matsuno-Yagi, Akemi, Yagi, Takao
Publicado 2011

Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans

por Gernoux, Gwladys, Gruntman, Alisha M., Blackwood, Meghan, Zieger, Marina, Flotte, Terence R., Mueller, Christian
Publicado 2020

Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice

por Zieger, Marina, Borel, Florie, Greer, Cynthia, Gernoux, Gwladys, Blackwood, Meghan, Flotte, Terence R., Mueller, Christian
Publicado 2022

Secretion of functional α1-antitrypsin is cell type dependent: Implications for intramuscular delivery for gene therapy

por Ke, Haiping, Guay, Kevin P., Flotte, Terence R., Gierasch, Lila M., Gershenson, Anne, Hebert, Daniel N.
Publicado 2022

Common pathways to Dean of Medicine at U.S. medical schools

por Jacobson, Clare E., Beeler, Whitney H., Griffith, Kent A., Flotte, Terence R., Byington, Carrie L., Jagsi, Reshma
Publicado 2021

In vitro and in vivo Functional Characterization of Gutless Recombinant SV40-derived CFTR Vectors

por Mueller, Christian, Strayer, Marlene S, Sirninger, Jeffery, Braag, Sofia, Branco, Francisco, Louboutin, Jean-Pierre, Flotte, Terence R., Strayer, David S.
Publicado 2009

Sustained miRNA-mediated Knockdown of Mutant AAT With Simultaneous Augmentation of Wild-type AAT Has Minimal Effect on Global Liver miRNA Profiles

por Mueller, Christian, Tang, Qiushi, Gruntman, Alisha, Blomenkamp, Keith, Teckman, Jeffery, Song, Lina, Zamore, Phillip D, Flotte, Terence R
Publicado 2012

Two-Plasmid Packaging System for Recombinant Adeno-Associated Virus

por Tang, Qiushi, Keeler, Allison M., Zhang, Songbo, Su, Qin, Lyu, Zhuoyao, Cheng, Yangfan, Gao, Guangping, Flotte, Terence R.
Publicado 2020

Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells

por Arjomandnejad, Motahareh, Sylvia, Katelyn, Blackwood, Meghan, Nixon, Thomas, Tang, Qiushi, Muhuri, Manish, Gruntman, Alisha M., Gao, Guangping, Flotte, Terence R., Keeler, Allison M.
Publicado 2021

Long-term Correction of Very Long-chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy

por Keeler, Allison M, Conlon, Thomas, Walter, Glenn, Zeng, Huadong, Shaffer, Scott A, Dungtao, Fu, Erger, Kirsten, Cossette, Travis, Tang, Qiushi, Mueller, Christian, Flotte, Terence R
Publicado 2012

Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency

por Borel, Florie, Tang, Qiushi, Gernoux, Gwladys, Greer, Cynthia, Wang, Ziqiong, Barzel, Adi, Kay, Mark A., Shultz, Leonard D., Greiner, Dale L., Flotte, Terence R., Brehm, Michael A., Mueller, Christian
Publicado 2017

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

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

Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema

por Borel, Florie, Sun, Huaming, Zieger, Marina, Cox, Andrew, Cardozo, Brynn, Li, Weiying, Oliveira, Gabriella, Davis, Airiel, Gruntman, Alisha, Flotte, Terence R., Brodsky, Michael H., Hoffman, Andrew M., Elmallah, Mai K., Mueller, Christian
Publicado 2018

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

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

Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study

por Gruntman, Alisha M., Gernoux, Gwladys, Tang, Qiushi, Ye, Guo-Jie, Knop, Dave R., Wang, Gensheng, Benson, Janet, Coleman, Kristen E., Keeler, Allison M., Mueller, Christian, Chicoine, Louis G., Chulay, Jeffrey D., Flotte, Terence R.
Publicado 2019

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

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