المؤلفون: Truong, Brian, Allegri, Gabriella, Liu, Xiao-Bo, Burke, Kristine E, Zhu, Xuling, Cederbaum, Stephen D, Häberle, Johannes, Martini, Paolo GV, Lipshutz, Gerald S

المصدر: Proceedings of the National Academy of Sciences of the United States of America. 116(42)

مصطلحات موضوعية: Medical Biotechnology, Biomedical and Clinical Sciences, Rare Diseases, Biotechnology, Nanotechnology, Digestive Diseases, Chronic Liver Disease and Cirrhosis, Nutrition, Bioengineering, Gene Therapy, Liver Disease, Brain Disorders, Genetics, Development of treatments and therapeutic interventions, 5.1 Pharmaceuticals, 5.2 Cellular and gene therapies, Oral and gastrointestinal, Ammonia, Animals, Arginase, Arginine, Codon, Disease Models, Animal, Glutamine, Hyperammonemia, Hyperargininemia, Lipids, Liver, Liver Diseases, Male, Mice, Mice, Inbred C57BL, Nanoparticles, RNA, Messenger, Urea, arginase deficiency, hyperargininemia, lipid nanoparticle, mRNA, ureagenesis

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/5dd780zxTest

المؤلفون: Cederbaum, Stephen D., Edwards, Jeffrey, Kellmeyer, Terrie, Peters, Yvette, Steiner, Robert D.

المصدر: Molecular Genetics and Metabolism ; volume 138, issue 4, page 107558 ; ISSN 1096-7192

مصطلحات موضوعية: Endocrinology, Genetics, Molecular Biology, Biochemistry, Endocrinology, Diabetes and Metabolism

الإتاحة: https://doi.org/10.1016/j.ymgme.2023.107558Test
https://api.elsevier.com/content/article/PII:S1096719223001889?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S1096719223001889?httpAccept=text/plainTest

المؤلفون: Angarita, Stephanie AK, Truong, Brian, Khoja, Suhail, Nitzahn, Matthew, Rajbhandari, Abha K, Zhuravka, Irina, Duarte, Sergio, Lin, Michael G, Lam, Alex K, Cederbaum, Stephen D, Lipshutz, Gerald S

المصدر: Molecular Genetics and Metabolism. 124(2)

مصطلحات موضوعية: Medical Biotechnology, Biomedical and Clinical Sciences, Genetics, Transplantation, Chronic Liver Disease and Cirrhosis, Liver Disease, Nutrition, Rare Diseases, Digestive Diseases, Development of treatments and therapeutic interventions, 5.2 Cellular and gene therapies, Oral and gastrointestinal, Animals, Arginase, Cells, Cultured, Disease Models, Animal, Female, Genetic Predisposition to Disease, Hepatocytes, Humans, Liver Diseases, Male, Metabolic Diseases, Mice, Mice, Knockout, Arginase deficiency, Hyperargininemia, Ammonia, Urea cycle disorder, Treatment, Cellular transplant, Clinical Sciences, Genetics & Heredity, Clinical sciences

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/7mx9b71dTest

المؤلفون: Lee, Patrick C, Truong, Brian, Vega-Crespo, Agustin, Gilmore, W Blake, Hermann, Kip, Angarita, Stephanie Ak, Tang, Jonathan K, Chang, Katherine M, Wininger, Austin E, Lam, Alex K, Schoenberg, Benjamen E, Cederbaum, Stephen D, Pyle, April D, Byrne, James A, Lipshutz, Gerald S

المصدر: Molecular therapy. Nucleic acids. 5(11)

مصطلحات موضوعية: arginase, genomic addition, hepatocytes, PSCs, urea cycle, Biochemistry and Cell Biology, Clinical Sciences

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/1vn3q67dTest

المؤلفون: Diaz, George A., Bechter, Mark, Cederbaum, Stephen D.

المساهمون: Aeglea BioTherapeutics

المصدر: Journal of Inherited Metabolic Disease ; volume 46, issue 1, page 3-14 ; ISSN 0141-8955 1573-2665

الإتاحة: https://doi.org/10.1002/jimd.12564Test

المؤلفون: 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.

المصدر: Molecular Therapy - Nucleic Acids ; volume 28, page 859-874 ; ISSN 2162-2531

مصطلحات موضوعية: Drug Discovery, Molecular Medicine

الإتاحة: https://doi.org/10.1016/j.omtn.2022.04.012Test
https://api.elsevier.com/content/article/PII:S2162253122000889?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2162253122000889?httpAccept=text/plainTest

المؤلفون: Cantero, Gloria, Liu, Xiao-Bo, Mervis, Ronald F, Lazaro, Maria T, Cederbaum, Stephen D, Golshani, Peyman, Lipshutz, Gerald S

المصدر: Journal of Neuroscience. 36(25)

مصطلحات موضوعية: Biomedical and Clinical Sciences, Neurosciences, Neurodegenerative, Gene Therapy, Pediatric, Genetics, Brain Disorders, Neurological, Action Potentials, Ammonia, Animals, Animals, Newborn, Arginase, Disease Models, Animal, Genetic Therapy, Hyperargininemia, In Vitro Techniques, Mice, Mice, Transgenic, Motor Cortex, Nerve Net, Neurons, Picrotoxin, Recovery of Function, Sodium Channel Blockers, Synapses, Tetrodotoxin, adeno-associated virus, arginase deficiency, electrophysiology, gene therapy, motor cortex, synapses, Medical and Health Sciences, Psychology and Cognitive Sciences, Neurology & Neurosurgery

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/8wb7449pTest

المؤلفون: Cederbaum, Stephen D.

المصدر: Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics ; page xv-lxiii ; ISBN 9780128125373

الإتاحة: https://doi.org/10.1016/b978-0-12-812537-3.02001-1Test
https://api.elsevier.com/content/article/PII:B9780128125373020011?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:B9780128125373020011?httpAccept=text/plainTest

المؤلفون: Antonarakis, Stylianos E., Beasley, T. Mark, Butcher, Darci T., Calais-Ferreira, Lucas, Cantor, Rita M., Cederbaum, Stephen D., Choufani, Sanaa, Cook, Jackie, Cooper, David N., Craig, Jeffrey M., Crowley, Michael R., Cytrynbaum, Cheryl, Fialkowski, Allison, Ginsburg, Geoffrey S., Grody, Wayne W., Haga, Susanne B., Hall, Judith G., Hegde, Madhuri R., Hisama, Fuki M., Johnston, H. Richard, Keats, Bronya J.B., Korf, Bruce R., Lott, Marie T., Martin, George M., Mikhail, Fady M., Nebert, Daniel W., Oshima, Junko, Procaccio, Vincent, Pyeritz, Reed E., Scurrah, Katrina J., Sherman, Stephanie L., Siu, Michelle T., Tiwari, Hemant K., Tycko, Benjamin, Umstad, Mark P., Wallace, Douglas C., Weksberg, Rosanna, Zhang, Ge

المصدر: Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics ; page ix-x ; ISBN 9780128125373

الإتاحة: https://doi.org/10.1016/b978-0-12-812537-3.01002-7Test
https://api.elsevier.com/content/article/PII:B9780128125373010027?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:B9780128125373010027?httpAccept=text/plainTest

المؤلفون: 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

مصطلحات موضوعية: Gene therapy, Genetic diseases, Neurodevelopment, Neuroscience, Animals, Arginase, Axons, Central Nervous System, Disease Models, Animal, Female, Gene Expression Profiling, Gene Expression Regulation, Genetic Predisposition to Disease, Genetic Therapy, Homozygote, Hyperargininemia, Liver, Male, Mice, Knockout, Oligodendroglia, Transcriptome

العلاقة: http://hdl.handle.net/10668/14472Test; PMC6777909; http://insight.jci.org/articles/view/130260/files/pdfTest; https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6777909/pdfTest

الإتاحة: https://doi.org/10.1172/jci.insight.130260Test
http://hdl.handle.net/10668/14472Test
http://insight.jci.org/articles/view/130260/files/pdfTest
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6777909/pdfTest