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Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control Diep, Taryn; Zhou, Wesley; Reyes, Rachel E.; Nitzahn, Matthew; Day, Isabel L.; Makris, Georgios; Lueptow, Lindsay; Zhuravka, Irina; Bakshi, Stuti; Gangoiti, Jon; Padaon, Hyacinth; Li, Yunfeng; Barshop, Bruce A.; Haberle, Johannes; Lipshutz, Gerald S. Share Save
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Design of a Phase 3 study of AAV-mediated gene transfer of ornithine transcarbamylase (OTC) in patients with late-onset OTC deficiency Konczal, Laura; Baker, Joshua; Wagenmakers, Margreet; Arnoux, Jean Baptiste; Burrow, T. Andrew; Couce, Marie Luz; del Toro, Mireia; Guelbert, Norberto; Kleppe, Soledad; Lipshutz, Gerald S.; Longo, Nicola; Matsumoto, Shiro; Schulze, Andreas; Thomas, Janet; Merritt, J. Lawrence, II Share Save
Arrested development of oligodendrocytes by guanidino compounds, not arginine, fosters dysmyelination in arginase deficiency Diep, Taryn; Zhang, Lizzie; Le, Colleen; Liu, Xiao-Bo; Patel, Puja; Bakshi, Stuti; Tkachyova, Ilona; Schulze, Andreas; Lipshutz, Gerald S. Share Save
Physical Frailty Predicts Outcomes in Patients Undergoing Evaluation for Kidney Transplantation Schaenman, Joanna; Ahn, Richard; Lee, Christine; Hale-Durbin, Bethany; Abdalla, Basmah; Danovitch, Gabriel; Huynh, Alina; Laviolette, Robert; Shigri, Ahson; Bunnapradist, Suphamai; Kendrick, Elizabeth; Lipshutz, Gerald S.; Pham, Phuong-Thu; Lum, Erik L.; Yabu, Julie M.; Seligman, Ben; Goldwater, Deena Share Save
Transforming the Future of Surgeon-Scientists Ladner, Daniela P.; Goldstein, Allan M.; Billiar, Timothy R.; Cameron, Andrew M.; Carpizo, Darren R.; Chu, Daniel I.; Coopersmith, Craig M.; Dematteo, Ronald P.; Feng, Sandy; Gallagher, Katherine A.; Gillanders, William E.; Lal, Brajesh K.; Lipshutz, Gerald S.; Liu, Annie; Maier, Ronald V.; Mittendorf, Elizabeth A.; Morris, Arden M.; Sicklick, Jason K.; Velazquez, Omaida C.; Whitson, Bryan A.; Wilke, Lee G.; Yoon, Sam S.; Zeiger, Martha A.; Farmer, Diana L.; Hwang, E. Shelley Share Save
DESIGN OF A PHASE 3 STUDY OF AAV-MEDIATED GENE TRANSFER OF ORNITHINE TRANSCARBAMYLASE (OTC) IN PATIENTS WITH LATE-ONSET OTC DEFICIENCY Konczal, Laura; Luz Couce, Marie; del Toro, Mireia; Langendonk, Janneke; Schulze, Andreas; Baker, Joshua; Burrow, T. Andrew; Lipshutz, Gerald S.; Longo, Nicola; Thomas, Janet; Merritt, J. Lawrence, II Share Save
O-GlcNAcylation enhances CPS1 catalytic efficiency for ammonia and promotes ureagenesis Soria, Leandro R.; Makris, Georgios; D'Alessio, Alfonso M.; De Angelis, Angela; Boffa, Iolanda; Pravata, Veronica M.; Rufenacht, Veronique; Attanasio, Sergio; Nusco, Edoardo; Arena, Paola; Ferenbach, Andrew T.; Paris, Debora; Cuomo, Paola; Motta, Andrea; Nitzahn, Matthew; Lipshutz, Gerald S.; Martinez-Pizarro, Ainhoa; Richard, Eva; Desviat, Lourdes R.; Haeberle, Johannes; van Aalten, Daan M. F.; Brunetti-Pierri, Nicola Share Save
Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency Khoja, Suhail; Liu, Xiao-Bo; Truong, Brian; Nitzahn, Matthew; Lambert, Jenna; Eliav, Adam; Nasser, Eram; Randolph, Emma; Burke, Kristine E.; White, Rebecca; Zhu, Xuling; Martini, Paolo G. V.; Nissim, Itzhak; Cederbaum, Stephen D.; Lipshutz, Gerald S. Share Save
Targeted deletion of PAC1 receptors in retinal neurons enhances neuron loss and axonopathy in a model of multiple sclerosis and optic neuritis Van, Christina; Condro, Michael C.; Ko, Henly H.; Hoang, Anh Q.; Zhu, Ruoyan; Lov, Kenny; Ricaflanca, Patrick T.; Diep, Anna L.; Nguyen, Nhat N. M.; Lipshutz, Gerald S.; MacKenzie-Graham, Allan; Waschek, James A. Share Save
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Hepatic arginase deficiency fosters dysmyelination during postnatal CNS development Liu, Xiao-Bo; Haney, Jillian R.; Cantero, Gloria; Lambert, Jenna R.; Otero-Garcia, Marcos; Truong, Brian; Gropman, Andrea; Cobos, Inma; Cederbaum, Stephen D.; Lipshutz, Gerald S. Share Save
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