Age-Related Accumulation of Somatic Mitochondrial DNA Mutations in Adult-Derived Human iPSCs
Kang, Eunju, Wang, Xinjian, Tippner-Hedges, Rebecca, Ma, Hong, Folmes, Clifford D.L., Gutierrez, Nuria Marti, Lee, Yeonmi, Van Dyken, Crystal, Ahmed, Riffat, Li, Ying, Koski, Amy, Hayama, Tomonari, Luo, Shiyu, Harding, Cary O., Amato, Paula, Jensen, Jeffrey, Battaglia, David, Lee, David, Wu, Diana, Terzic, Andre, Wolf, Don P., Huang, Taosheng, Mitalipov, Shoukhrat
Published in Cell stem cell (05.05.2016)
Published in Cell stem cell (05.05.2016)
Get full text
Journal Article
Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: an open-label, multicentre, phase 1 dose-escalation trial
Longo, Nicola, MD, Harding, Cary O, MD, Burton, Barbara K, MD, Grange, Dorothy K, MD, Vockley, Jerry, MD, Wasserstein, Melissa, MD, Rice, Gregory M, MD, Dorenbaum, Alejandro, MD, Neuenburg, Jutta K, MD, Musson, Donald G, PhD, Gu, Zhonghua, PhD, Sile, Saba, MD
Published in The Lancet (British edition) (05.07.2014)
Published in The Lancet (British edition) (05.07.2014)
Get full text
Journal Article
Triheptanoin versus trioctanoin for long-chain fatty acid oxidation disorders: a double blinded, randomized controlled trial
Gillingham, Melanie B., Heitner, Stephen B., Martin, Julie, Rose, Sarah, Goldstein, Amy, El-Gharbawy, Areeg Hassan, Deward, Stephanie, Lasarev, Michael R., Pollaro, Jim, DeLany, James P., Burchill, Luke J., Goodpaster, Bret, Shoemaker, James, Matern, Dietrich, Harding, Cary O., Vockley, Jerry
Published in Journal of inherited metabolic disease (01.11.2017)
Published in Journal of inherited metabolic disease (01.11.2017)
Get full text
Journal Article
Treatment of phenylketonuria using minicircle‐based naked‐DNA gene transfer to murine liver
Viecelli, Hiu Man, Harbottle, Richard P., Wong, Suet Ping, Schlegel, Andrea, Chuah, Marinee K., VandenDriessche, Thierry, Harding, Cary O., Thöny, Beat
Published in Hepatology (Baltimore, Md.) (01.09.2014)
Published in Hepatology (Baltimore, Md.) (01.09.2014)
Get full text
Journal Article
Meta-analysis of bone mineral density in adults with phenylketonuria
Rocha, Júlio C, Hermida, Álvaro, Jones, Cheryl J, Wu, Yunchou, Clague, Gillian E, Rose, Sarah, Whitehall, Kaleigh B, Ahring, Kirsten K, Pessoa, André L S, Harding, Cary O, Rohr, Fran, Inwood, Anita, Longo, Nicola, Muntau, Ania C, Sivri, Serap, Maillot, François
Published in Orphanet journal of rare diseases (12.09.2024)
Published in Orphanet journal of rare diseases (12.09.2024)
Get full text
Journal Article
Systematic literature review of the somatic comorbidities experienced by adults with phenylketonuria
Whitehall, Kaleigh B, Rose, Sarah, Clague, Gillian E, Ahring, Kirsten K, Bilder, Deborah A, Harding, Cary O, Hermida, Álvaro, Inwood, Anita, Longo, Nicola, Maillot, François, Muntau, Ania C, Pessoa, André L S, Rocha, Júlio C, Rohr, Fran, Sivri, Serap, Said, Jack, Oshinbolu, Sheun, Sibbring, Gillian C
Published in Orphanet journal of rare diseases (12.08.2024)
Published in Orphanet journal of rare diseases (12.08.2024)
Get full text
Journal Article
Pharmacologic inhibition of L-tyrosine degradation ameliorates cerebral dopamine deficiency in murine phenylketonuria (PKU)
Harding, Cary O., Winn, Shelley R., Gibson, K. Michael, Arning, Erland, Bottiglieri, Teodoro, Grompe, Markus
Published in Journal of inherited metabolic disease (01.09.2014)
Published in Journal of inherited metabolic disease (01.09.2014)
Get full text
Journal Article
Expert Consensus on the Long-Term Effectiveness of Medical Nutrition Therapy and Its Impact on the Outcomes of Adults with Phenylketonuria
Rocha, Júlio César, Ahring, Kirsten K, Bausell, Heather, Bilder, Deborah A, Harding, Cary O, Inwood, Anita, Longo, Nicola, Muntau, Ania C, Pessoa, André L. Santos, Rohr, Fran, Sivri, Serap, Hermida, Álvaro
Published in Nutrients (01.09.2023)
Published in Nutrients (01.09.2023)
Get full text
Journal Article
Nutrient intake, body composition, and blood phenylalanine control in children with phenylketonuria compared to healthy controls
Sailer, Melissa, Elizondo, Gabriela, Martin, Julie, Harding, Cary O., Gillingham, Melanie B.
Published in Molecular genetics and metabolism reports (01.06.2020)
Published in Molecular genetics and metabolism reports (01.06.2020)
Get full text
Journal Article
Development of a porcine model of phenylketonuria with a humanized R408W mutation for gene editing
Kaiser, Robert A, Carlson, Daniel F, Allen, Kari L, Webster, Dennis A, VanLith, Caitlin J, Nicolas, Clara T, Hillin, Lori G, Yu, Yue, Kaiser, Catherine W, Wahoff, William R, Hickey, Raymond D, Watson, Adrienne L, Winn, Shelley R, Thöny, Beat, Kern, Douglas R, Harding, Cary O, Lillegard, Joseph B
Published in PloS one (25.01.2021)
Published in PloS one (25.01.2021)
Get full text
Journal Article
Autologous Gene and Cell Therapy Provides Safe and Long-Term Curative Therapy in A Large Pig Model of Hereditary Tyrosinemia Type 1
Hickey, Raymond D., Nicolas, Clara T., Allen, Kari, Mao, Shennen, Elgilani, Faysal, Glorioso, Jaime, Amiot, Bruce, VanLith, Caitlin, Guthman, Rebekah, Du, Zeji, Chen, Harvey, Harding, Cary O., Kaiser, Robert A., Nyberg, Scott L., Lillegard, Joseph B.
Published in Cell transplantation (01.01.2019)
Published in Cell transplantation (01.01.2019)
Get full text
Journal Article
Hepatocyte-targeted HFE and TFR2 control hepcidin expression in mice
Gao, Junwei, Chen, Juxing, De Domenico, Ivana, Koeller, David M., Harding, Cary O., Fleming, Robert E., Koeberl, Dwight D., Enns, Caroline A.
Published in Blood (22.04.2010)
Published in Blood (22.04.2010)
Get full text
Journal Article
Pharmacokinetic, pharmacodynamic, and immunogenic rationale for optimal dosing of pegvaliase, a PEGylated bacterial enzyme, in adult patients with phenylketonuria
Qi, Yulan, Patel, Gina, Henshaw, Joshua, Gupta, Soumi, Olbertz, Joy, Larimore, Kevin, Harding, Cary O., Merilainen, Markus, Zori, Roberto, Longo, Nicola, Burton, Barbara K., Li, Mingjin, Gu, Zhonghua, Zoog, Stephen J., Weng, Haoling H., Schweighardt, Becky
Published in Clinical and translational science (01.09.2021)
Published in Clinical and translational science (01.09.2021)
Get full text
Journal Article
Pegvaliase for the treatment of phenylketonuria: Final results of a long-term phase 3 clinical trial program
Harding, Cary O., Longo, Nicola, Northrup, Hope, Sacharow, Stephanie, Singh, Rani, Thomas, Janet A., Vockley, Jerry, Zori, Roberto T., Bulloch Whitehall, Kaleigh, Lilienstein, Joshua, Lindstrom, Kristin, Levy, Drew G., Jones, Shaun, Burton, Barbara K.
Published in Molecular genetics and metabolism reports (01.06.2024)
Published in Molecular genetics and metabolism reports (01.06.2024)
Get full text
Journal Article
Infants suspected to have very-long chain acyl-CoA dehydrogenase deficiency from newborn screening
Merritt, J. Lawrence, Vedal, Sverre, Abdenur, Jose E., Au, Sylvia M., Barshop, Bruce A., Feuchtbaum, Lisa, Harding, Cary O., Hermerath, Cheryl, Lorey, Fred, Sesser, David E., Thompson, John D., Yu, Arthur
Published in Molecular genetics and metabolism (01.04.2014)
Published in Molecular genetics and metabolism (01.04.2014)
Get full text
Journal Article
A G1528C Hadha knock-in mouse model recapitulates aspects of human clinical phenotypes for long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency
Gaston, Garen, Babcock, Shannon, Ryals, Renee, Elizondo, Gabriela, DeVine, Tiffany, Wafai, Dahlia, Packwood, William, Holden, Sarah, Raber, Jacob, Lindner, Jonathan R, Pennesi, Mark E, Harding, Cary O, Gillingham, Melanie B
Published in Communications biology (29.08.2023)
Published in Communications biology (29.08.2023)
Get full text
Journal Article