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Molecular Therapy. Nucleic Acids
|
January 22, 2026
Enhancement of therapeutic transgene insertion for treatment of murine phenylketonuria
Michael A Martinez, Daelyn Y Richards, Shelley R Winn, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
March 19, 2024
Cardiac phenotype in adolescents and young adults with long-chain 3-hydroxyacyl CoA dehydrogenase (LCHAD) deficiency
Gabriela Elizondo, Ajesh Saini, Cesar Gonzalez de Alba, et al.
Ophthalmic Genetics
|
March 17, 2016
Spectrum of ocular manifestations in cobalamin C and cobalamin A types of methylmalonic acidemia
Cristy A Ku, Jacqueline K Ng, Daniel J Karr, et al.
Molecular Genetics and Metabolism
|
October 28, 2011
Substrate oxidation and cardiac performance during exercise in disorders of long chain fatty acid oxidation
Annie M Behrend, Cary O Harding, James D Shoemaker, et al.
Journal of Perinatology : Official Journal of the California Perinatal Association
|
July 9, 2003
Potential for misdiagnosis due to lack of metabolic derangement in combined methylmalonic aciduria/hyperhomocysteinemia (cblC) in the neonate
Cary O Harding, De-Ann M Pillers, Robert D Steiner, et al.
Molecular Genetics and Metabolism
|
March 27, 2022
Modeling the cognitive effects of diet discontinuation in adults with phenylketonuria (PKU) using pegvaliase therapy in PAH-deficient mice
Shelley R Winn, Sandra Dudley, Tanja Scherer, et al.
Hepatology (Baltimore, Md.)
|
March 4, 2014
Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver
Hiu Man Viecelli, Richard P Harbottle, Suet Ping Wong, et al.
Molecular Genetics and Metabolism
|
November 16, 2025
Measuring dietary intake among participants with a urea cycle disorder using standard diet records or a novel food photography app
Elaine Sim, Ashley Gregor, Erin MacLeod, et al.
Blood
|
February 24, 2010
Hepatocyte-targeted HFE and TFR2 control hepcidin expression in mice
Junwei Gao, Juxing Chen, Ivana De Domenico, et al.
Paediatric Drugs
|
November 1, 2019
Long-Term Outcomes and Practical Considerations in the Pharmacological Management of Tyrosinemia Type 1
Willem G van Ginkel, Iris L Rodenburg, Cary O Harding, et al.
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of 12
Search research articles
Search
Showing results (51-60 of 113) with videos related to
Sort By:
Page
of 12
Molecular Therapy. Nucleic Acids
|
January 22, 2026
Enhancement of therapeutic transgene insertion for treatment of murine phenylketonuria
Michael A Martinez, Daelyn Y Richards, Shelley R Winn, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics
|
March 19, 2024
Cardiac phenotype in adolescents and young adults with long-chain 3-hydroxyacyl CoA dehydrogenase (LCHAD) deficiency
Gabriela Elizondo, Ajesh Saini, Cesar Gonzalez de Alba, et al.
Ophthalmic Genetics
|
March 17, 2016
Spectrum of ocular manifestations in cobalamin C and cobalamin A types of methylmalonic acidemia
Cristy A Ku, Jacqueline K Ng, Daniel J Karr, et al.
Molecular Genetics and Metabolism
|
October 28, 2011
Substrate oxidation and cardiac performance during exercise in disorders of long chain fatty acid oxidation
Annie M Behrend, Cary O Harding, James D Shoemaker, et al.
Journal of Perinatology : Official Journal of the California Perinatal Association
|
July 9, 2003
Potential for misdiagnosis due to lack of metabolic derangement in combined methylmalonic aciduria/hyperhomocysteinemia (cblC) in the neonate
Cary O Harding, De-Ann M Pillers, Robert D Steiner, et al.
Molecular Genetics and Metabolism
|
March 27, 2022
Modeling the cognitive effects of diet discontinuation in adults with phenylketonuria (PKU) using pegvaliase therapy in PAH-deficient mice
Shelley R Winn, Sandra Dudley, Tanja Scherer, et al.
Hepatology (Baltimore, Md.)
|
March 4, 2014
Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver
Hiu Man Viecelli, Richard P Harbottle, Suet Ping Wong, et al.
Molecular Genetics and Metabolism
|
November 16, 2025
Measuring dietary intake among participants with a urea cycle disorder using standard diet records or a novel food photography app
Elaine Sim, Ashley Gregor, Erin MacLeod, et al.
Blood
|
February 24, 2010
Hepatocyte-targeted HFE and TFR2 control hepcidin expression in mice
Junwei Gao, Juxing Chen, Ivana De Domenico, et al.
Paediatric Drugs
|
November 1, 2019
Long-Term Outcomes and Practical Considerations in the Pharmacological Management of Tyrosinemia Type 1
Willem G van Ginkel, Iris L Rodenburg, Cary O Harding, et al.
Page
of 12