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Number of items: 8.

Winn, Shelley R; Scherer, Tanja; Thöny, Beat; Ying, Ming; Martinez, Aurora; Weber, Sydney; Raber, Jacob; Harding, Cary O (2018). Blood phenylalanine reduction corrects CNS dopamine and serotonin deficiencies and partially improves behavioral performance in adult phenylketonuric mice. Molecular Genetics and Metabolism, 123(1):6-20.

Grisch-Chan, Hiu Man; Schlegel, Andrea; Scherer, Tanja; Allegri, Gabriella; Heidelberger, Raphael; Tsikrika, Panagiota; Schmeer, Marco; Schleef, Martin; Harding, Cary O; Häberle, Johannes; Thöny, Beat (2017). Low-dose gene therapy for murine PKU using episomal naked DNA vectors expressing PAH from Its endogenous liver promoter. Molecular Therapy - Nucleic Acids, 7:339-349.

Winn, Shelley R; Scherer, Tanja; Thöny, Beat; Harding, Cary O (2016). High dose sapropterin dihydrochloride therapy improves monoamine neurotransmitter turnover in murine phenylketonuria (PKU). Molecular Genetics and Metabolism, 117(1):5-11.

Lee, Brendan; Diaz, George A; Rhead, William; Lichter-Konecki, Uta; Feigenbaum, Annette; Berry, Susan A; Le Mons, Cindy; Bartley, James A; Longo, Nicola; Nagamani, Sandesh C; Berquist, William; Gallagher, Renata; Bartholomew, Dennis; Harding, Cary O; Korson, Mark S; McCandless, Shawn E; Smith, Wendy; Cederbaum, Stephen; Wong, Derek; Merritt, J Lawrence; Schulze, Andreas; Vockley, Jerry; Vockley, Gerard; Kronn, David; Zori, Roberto; Summar, Marshall; Milikien, Douglas A; Marino, Miguel; Coakley, Dion F; Mokhtarani, Masoud; Scharschmidt, Bruce F (2015). Blood ammonia and glutamine as predictors of hyperammonemic crises in patients with urea cycle disorder. Genetics in Medicine, 17(7):561-568.

Viecelli, Hiu Man; Harbottle, Richard P; Wong, Suet Ping; Schlegel, Andrea; Chuah, Marinee K; Van den Driessche, Thierry; Harding, Cary O; Thöny, Beat (2014). Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver. Hepatology, 60(3):1035-1043.

Camp, Kathryn M; Parisi, Melissa A; Acosta, Phyllis B; Berry, Gerard T; Bilder, Deborah A; Blau, Nenad; Bodamer, Olaf A; Brosco, Jeffrey P; Brown, Christine S; Burlina, Alberto B; Burton, Barbara K; Chang, Christine S; Coates, Paul M; Cunningham, Amy C; Dobrowolski, Steven F; Ferguson, John H; Franklin, Thomas D; Frazier, Dianne M; Grange, Dorothy K; Greene, Carol L; Groft, Stephen C; Harding, Cary O; Howell, R Rodney; Huntington, Kathleen L; Hyatt-Knorr, Henrietta D; Jevaji, Indira P; Levy, Harvey L; Lichter-Konecki, Uta; Lindegren, Mary Lou; Lloyd-Puryear, Michele A; Matalon, Kimberlee; MacDonald, Anita; McPheeters, Melissa L; Mitchell, John J; Mofidi, Shideh; Moseley, Kathryn D; Mueller, Christine M; Mulberg, Andrew E; Nerurkar, Lata S; Ogata, Beth N; Pariser, Anne R; Prasad, Suyash; Pridjian, Gabriella; Rasmussen, Sonja A; Reddy, Uma M; Rohr, Frances J; Singh, Rani H; Sirrs, Sandra M; Stremer, Stephanie E; Tagle, Danilo A; Thompson, Susan M; Urv, Tiina K; Utz, Jeanine R; van Spronsen, Francjan; Vockley, Jerry; Waisbren, Susan E; Weglicki, Linda S; White, Desirée A; Whitley, Chester B; Wilfond, Benjamin S; Yannicelli, Steven; Young, Justin M (2014). Phenylketonuria scientific review Conference: State of the science and future research needs. Molecular Genetics and Metabolism, 112(2):87-122.

Gallego-Villar, Lorena; Viecelli, Hiu Man; Pérez, Belén; Harding, Cary O; Ugarte, Magdalena; Thöny, Beat; Desviat, Lourdes R (2014). A sensitive assay system to test antisense oligonucleotides for splice suppression therapy in the mouse liver. Molecular Therapy - Nucleic Acids, 3:e193.

Harding, Cary O; Thöny, Beat (2011). Update on the development of gene therapy for PKU. National PKU News, 22(3):2-5.

This list was generated on Wed Jan 16 12:56:48 2019 CET.