Bibliotecas PUC-SP - Busca Integrada

Less is more: Allele-specific removal of dysfunctional RYR1 channel subunits

von Derek Sun, William R. Lagor
Veröffentlicht in Molecular Therapy: Nucleic Acids (2024-09-01)

Simple improvements in vector design afford substantial gains in AAV delivery of aggregation-slowing Aβ variants

von Ella Borgenheimer, Cameron Trueblood, Bryan L. Nguyen, William R. Lagor, Joanna L. Jankowsky
Veröffentlicht in Molecular Therapy: Nucleic Acids (2024-12-01)

Adeno-Associated virus 8 delivers an immunomodulatory peptide to mouse liver more efficiently than to rat liver

von Yuqing Wang, Ayrea Hurley, Marco De Giorgi, Mark R. Tanner, Rong-Chi Hu, Michael W. Pennington, William R. Lagor, Christine Beeton
Veröffentlicht in PLoS ONE (2023-01-01)

MBNL overexpression rescues cardiac phenotypes in a myotonic dystrophy type 1 heart mouse model

von Rong-Chi Hu, Yi Zhang, Larissa Nitschke, Sara J. Johnson, Ayrea E. Hurley, William R. Lagor, Zheng Xia, Thomas A. Cooper
Veröffentlicht in The Journal of Clinical Investigation (2025-04-01)

Adeno-Associated virus 8 delivers an immunomodulatory peptide to mouse liver more efficiently than to rat liver.

von Yuqing Wang, Ayrea Hurley, Marco De Giorgi, Mark R Tanner, Rong-Chi Hu, Michael W Pennington, William R Lagor, Christine Beeton
Veröffentlicht in PLoS ONE (2023-01-01)

In vivo editing of the pan-endothelium by immunity evading simian adenoviral vector

von Reka Lorincz, Aluet Borrego Alvarez, Christopher J. Walkey, Samir A. Mendonça, Zhi Hong Lu, Alexa E. Martinez, Cecilia Ljungberg, Jason D. Heaney, William R. Lagor, David T. Curiel
Veröffentlicht in Biomedicine & Pharmacotherapy (2023-02-01)

Overexpression and deletion of phospholipid transfer protein reduce HDL mass and cholesterol efflux capacity but not macrophage reverse cholesterol transport

von Takashi Kuwano, Xin Bi, Eleonora Cipollari, Tomoyuki Yasuda, William R. Lagor, Hannah J. Szapary, Junichiro Tohyama, John S. Millar, Jeffrey T. Billheimer, Nicholas N. Lyssenko, Daniel J. Rader
Veröffentlicht in Journal of Lipid Research (2017-04-01)

In vivo genome editing at the albumin locus to treat methylmalonic acidemia

von Jessica L. Schneller, Ciaran M. Lee, Leah E. Venturoni, Randy J. Chandler, Ang Li, Sangho Myung, Thomas J. Cradick, Ayrea E. Hurley, William R. Lagor, Gang Bao, Charles P. Venditti
Veröffentlicht in Molecular Therapy: Methods & Clinical Development (2021-12-01)

A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing

von Ang Li, Ciaran M. Lee, Ayrea E. Hurley, Kelsey E. Jarrett, Marco De Giorgi, Weiqi Lu, Karol S. Balderrama, Alexandria M. Doerfler, Harshavardhan Deshmukh, Anirban Ray, Gang Bao, William R. Lagor
Veröffentlicht in Molecular Therapy: Methods & Clinical Development (2019-03-01)

Depletion of essential isoprenoids and ER stress induction following acute liver-specific deletion of HMG-CoA reductase

von Marco De Giorgi, Kelsey E. Jarrett, Jason C. Burton, Alexandria M. Doerfler, Ayrea Hurley, Ang Li, Rachel H. Hsu, Mia Furgurson, Kalyani R. Patel, Jun Han, Christoph H. Borchers, William R. Lagor
Veröffentlicht in Journal of Lipid Research (2020-12-01)

Targeting the Apoa1 locus for liver-directed gene therapy

von Marco De Giorgi, Ang Li, Ayrea Hurley, Mercedes Barzi, Alexandria M. Doerfler, Nikitha A. Cherayil, Harrison E. Smith, Jonathan D. Brown, Charles Y. Lin, Karl-Dimiter Bissig, Gang Bao, William R. Lagor
Veröffentlicht in Molecular Therapy: Methods & Clinical Development (2021-06-01)

The effects of apolipoprotein F deficiency on high density lipoprotein cholesterol metabolism in mice.

von William R Lagor, David W Fields, Sumeet A Khetarpal, Arthi Kumaravel, Wen Lin, Nathaniel Weintraub, Kaijin Wu, Sarah F Hamm-Alvarez, Denise Drazul-Schrader, Margarita de la Llera-Moya, George H Rothblat, Daniel J Rader
Veröffentlicht in PLoS ONE (2012-01-01)

TAp63 contributes to sexual dimorphism in POMC neuron functions and energy homeostasis

von Chunmei Wang, Yanlin He, Pingwen Xu, Yongjie Yang, Kenji Saito, Yan Xia, Xiaofeng Yan, Antentor Hinton Jr, Chunling Yan, Hongfang Ding, Likai Yu, Gang Shu, Rajat Gupta, Qi Wu, Qingchun Tong, William R. Lagor, Elsa R. Flores, Yong Xu
Veröffentlicht in Nature Communications (2018-04-01)

LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study

von Alexandria M. Doerfler, So Hyun Park, Julia M. Assini, Amer Youssef, Lavanya Saxena, Adam B. Yaseen, Marco De Giorgi, Marcel Chuecos, Ayrea E. Hurley, Ang Li, Santica M. Marcovina, Gang Bao, Michael B. Boffa, Marlys L. Koschinsky, William R. Lagor
Veröffentlicht in Molecular Therapy: Methods & Clinical Development (2022-12-01)

A chemical chaperone improves muscle function in mice with a RyR1 mutation

von Chang Seok Lee, Amy D. Hanna, Hui Wang, Adan Dagnino-Acosta, Aditya D. Joshi, Mark Knoblauch, Yan Xia, Dimitra K. Georgiou, Jianjun Xu, Cheng Long, Hisayuki Amano, Corey Reynolds, Keke Dong, John C. Martin, William R. Lagor, George G. Rodney, Ergun Sahin, Caroline Sewry, Susan L. Hamilton
Veröffentlicht in Nature Communications (2017-03-01)

TFEB regulates murine liver cell fate during development and regeneration

von Nunzia Pastore, Tuong Huynh, Niculin J. Herz, Alessia Calcagni’, Tiemo J. Klisch, Lorenzo Brunetti, Kangho Ho Kim, Marco De Giorgi, Ayrea Hurley, Annamaria Carissimo, Margherita Mutarelli, Niya Aleksieva, Luca D’Orsi, William R. Lagor, David D. Moore, Carmine Settembre, Milton J. Finegold, Stuart J. Forbes, Andrea Ballabio
Veröffentlicht in Nature Communications (2020-05-01)

Targeting AAV vectors to the central nervous system by engineering capsid-receptor interactions that enable crossing of the blood-brain barrier.

von Qin Huang, Albert T Chen, Ken Y Chan, Hikari Sorensen, Andrew J Barry, Bahar Azari, Qingxia Zheng, Thomas Beddow, Binhui Zhao, Isabelle G Tobey, Cynthia Moncada-Reid, Fatma-Elzahraa Eid, Christopher J Walkey, M Cecilia Ljungberg, William R Lagor, Jason D Heaney, Yujia A Chan, Benjamin E Deverman
Veröffentlicht in PLoS Biology (2023-07-01)

A human liver chimeric mouse model for non-alcoholic fatty liver disease

von Beatrice Bissig-Choisat, Michele Alves-Bezerra, Barry Zorman, Scott A. Ochsner, Mercedes Barzi, Xavier Legras, Diane Yang, Malgorzata Borowiak, Adam M. Dean, Robert B. York, N. Thao N. Galvan, John Goss, William R. Lagor, David D. Moore, David E. Cohen, Neil J. McKenna, Pavel Sumazin, Karl-Dimiter Bissig
Veröffentlicht in JHEP Reports (2021-06-01)

Speg interactions that regulate the stability of excitation-contraction coupling protein complexes in triads and dyads

von Chang Seok Lee, Sung Yun Jung, Rachel Sue Zhen Yee, Nadia H. Agha, Jin Hong, Ting Chang, Lyle W. Babcock, Jorie D. Fleischman, Benjamin Clayton, Amy D. Hanna, Christopher S. Ward, Denise Lanza, Ayrea E. Hurley, Pumin Zhang, Xander H. T. Wehrens, William R. Lagor, George G. Rodney, Susan L. Hamilton
Veröffentlicht in Communications Biology (2023-09-01)