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  • 2 Similar Profiles
Phenylketonurias Medicine & Life Sciences
Inborn Urea Cycle Disorder Medicine & Life Sciences
Phenylalanine Hydroxylase Medicine & Life Sciences
Phenylalanine Medicine & Life Sciences
Genetic Therapy Medicine & Life Sciences
Inborn Errors Metabolism Medicine & Life Sciences
3-Hydroxyacyl-CoA Dehydrogenase Medicine & Life Sciences
Liver Medicine & Life Sciences

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Projects 1976 2021

Phenylketonurias
Neurotransmitter Agents
Serotonin
Brain
Dopamine

Development and Characterization of a Swine Model of Phenylketonuria

Harding, C.

National Institutes of Health

6/1/165/31/17

Project: Research projectSmall Business Innovation Research Grants (SBIR) - Phase I

Phenylketonurias
Swine
Phenylalanine
Genetic Therapy
Therapeutics
Phenylketonurias
Phenylalanine
Muscles
Phenylalanine Hydroxylase
Therapeutics

Intravenous non-viral gene therapy for phenylketonuria (PKU)

Harding, C.

National Institutes of Health

9/20/069/19/08

Project: Research projectSmall Business Technology Transfer (STTR) Grants - Phase I

Phenylketonurias
Phenylalanine Hydroxylase
Phenylalanine
Genetic Therapy
Skeletal Muscle

Stem Cell-Mediated Liver Repopulation for Murine PKU

Harding, C.

National Institutes of Health

1/1/0511/30/07

Project: Research projectExploratory/Developmental Grants

Phenylketonurias
Stem Cells
Hematopoietic Stem Cells
Liver
Hepatocytes

Research Output 1990 2018

  • 2241 Citations
  • 29 h-Index
  • 97 Article
  • 4 Chapter

Biochemical markers and neuropsychological functioning in distal urea cycle disorders

Members Of The Urea Cycle Disorders Consortium Feb 8 2018 (Accepted/In press) In : Journal of Inherited Metabolic Disease. p. 1-11 11 p.

Research output: Contribution to journalArticle

Inborn Urea Cycle Disorder
Hyperargininemia
Biomarkers
Citrullinemia
Ammonia
1 Citations

Blood phenylalanine reduction corrects CNS dopamine and serotonin deficiencies and partially improves behavioral performance in adult phenylketonuric mice

Winn, S. R., Scherer, T., Thöny, B., Ying, M., Martinez, A., Weber, S., Raber, J. & Harding, C. O. Jan 1 2018 In : Molecular Genetics and Metabolism. 123, 1, p. 6-20 15 p.

Research output: Contribution to journalArticle

Prefabricated roofs
Phenylalanine
Dopamine
Serotonin
Central Nervous System
1 Citations

Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial

PRISM-2 Investigators Jan 1 2018 (Accepted/In press) In : Molecular Genetics and Metabolism.

Research output: Contribution to journalArticle

Phase III Clinical Trials
Phenylketonurias
Phenylalanine
Blood
Placebos
1 Citations

Pegvaliase for the treatment of phenylketonuria: Results of a long-term phase 3 clinical trial program (PRISM)

PRISM investigators Jan 1 2018 (Accepted/In press) In : Molecular Genetics and Metabolism.

Research output: Contribution to journalArticle

Phase III Clinical Trials
Phenylketonurias
Phenylalanine
Blood
Anaphylaxis

Tetrahydrobiopterin treatment reduces brain L-Phe but only partially improves serotonin in hyperphenylalaninemic ENU1/2 mice

Scherer, T., Allegri, G., Sarkissian, C. N., Ying, M., Grisch-Chan, H. M., Rassi, A., Winn, S. R., Harding, C. O., Martinez, A. & Thöny, B. Mar 8 2018 (Accepted/In press) In : Journal of Inherited Metabolic Disease. p. 1-10 10 p.

Research output: Contribution to journalArticle

Tyrosine 3-Monooxygenase
Phenylalanine
Serotonin
Phenylketonurias
Brain