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  • 2 Similar Profiles
Therapeutics Medicine & Life Sciences
Phenylketonurias Medicine & Life Sciences
Liver Medicine & Life Sciences
Inborn Urea Cycle Disorder Medicine & Life Sciences
Phenylalanine Medicine & Life Sciences
Mutation Medicine & Life Sciences
Phenylalanine Hydroxylase Medicine & Life Sciences
Fatty Acids Medicine & Life Sciences

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Projects 1976 2021

Phenylketonurias
Neurotransmitter Agents
Therapeutics
Brain
Serotonin

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
Therapeutics
Phenylalanine
Genetic Therapy
Phenylketonurias
Phenylalanine
Muscles
Therapeutics
Phenylalanine Hydroxylase

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
Genetic Therapy
Phenylalanine Hydroxylase
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
Liver
Hematopoietic Stem Cells
Growth

Research Output 1990 2017

  • 2079 Citations
  • 29 h-Index
  • 91 Article
  • 4 Chapter

Fatty acid β-oxidation defects

Harding, C. O. Jan 6 2017 Metabolic Diseases: Foundations of Clinical Management, Genetics, and Pathology. IOS Press, p. 155-189 35 p.

Research output: ResearchChapter

Fatty Acids
Therapeutics
Hypoglycemia
Enzymes
Inborn Errors Metabolism

Gene and cell therapy for inborn errors of metabolism

Harding, C. O. Jan 1 2017 Inherited Metabolic Diseases: A Clinical Approach. Springer Berlin Heidelberg, p. 155-171 17 p.

Research output: ResearchChapter

2 Citations

Low-Dose Gene Therapy for Murine PKU Using Episomal Naked DNA Vectors Expressing PAH from Its Endogenous Liver Promoter

Grisch-Chan, H. M., Schlegel, A., Scherer, T., Allegri, G., Heidelberger, R., Tsikrika, P., Schmeer, M., Schleef, M., Harding, C. O., Häberle, J. & Thöny, B. Jun 1 2017 In : Molecular Therapy - Nucleic Acids. 7, p. 339-349 11 p.

Research output: Research - peer-reviewArticle

Phenylalanine Hydroxylase
Phenylketonurias
Genetic Therapy
Liver
DNA

Safety and efficacy of glycerol phenylbutyrate for management of urea cycle disorders in patients aged 2months to 2years

Berry, S. A., Longo, N., Diaz, G. A., McCandless, S. E., Smith, W. E., Harding, C. O., Zori, R., Ficicioglu, C., Lichter-Konecki, U., Robinson, B. & Vockley, J. 2017 (Accepted/In press) In : Molecular Genetics and Metabolism.

Research output: Research - peer-reviewArticle

Inborn Urea Cycle Disorder
Safety
glycerol phenylbutyrate
Urea
Glutamine

Triheptanoin versus trioctanoin for long-chain fatty acid oxidation disorders: a double blinded, randomized controlled trial

Gillingham, M. B., Heitner, S. B., Martin, J., Rose, S., Goldstein, A., El-Gharbawy, A. H., Deward, S., Lasarev, M. R., Pollaro, J., DeLany, J. P., Burchill, L. J., Goodpaster, B., Shoemaker, J., Matern, D., Harding, C. O. & Vockley, J. Sep 4 2017 (Accepted/In press) In : Journal of Inherited Metabolic Disease. p. 1-13 13 p.

Research output: Research - peer-reviewArticle

Fatty Acids
Randomized Controlled Trials
triheptanoin
tricaprylin
Triglycerides