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Molecular Genetics and Metabolism
|
July 22, 2022
Biochemical and behavioural profile of NTBC treated Tyrosinemie type 1 mice
Willem G van Ginkel, Shelley R Winn, Sandra Dudley, et al.
Journal of Child Neurology
|
August 15, 2002
Friedreich's ataxia associated with mitochondrial myopathy: clinicopathologic report
Catherine L Gallagher, Andrew J Waclawik, Brad R Beinlich, et al.
Investigative Ophthalmology & Visual Science
|
September 16, 2024
iPSC-Derived LCHADD Retinal Pigment Epithelial Cells Are Susceptible to Lipid Peroxidation and Rescued by Transfection of a Wildtype AAV-HADHA Vector
Tiffany DeVine, Gabriela Elizondo, Garen Gaston, et al.
Science Translational Medicine
|
June 10, 2021
Therapeutic liver repopulation by transient acetaminophen selection of gene-modified hepatocytes
Anne Vonada, Amita Tiyaboonchai, Sean Nygaard, et al.
Molecular Genetics and Metabolism
|
January 7, 2024
Evaluating change in diet with pegvaliase treatment in adults with phenylketonuria: Analysis of phase 3 clinical trial data
Fran Rohr, Barbara Burton, Anne Dee, et al.
Molecular Genetics and Metabolism
|
February 28, 2026
Management of pegvaliase-related skin concerns: best practice recommendations using a modified Delphi approach
Alvaro Hermida Ameijeiras, Erika Vucko, Cary O Harding, et al.
Molecular Genetics and Metabolism
|
November 26, 2024
Corrigendum to "Evaluating change in diet with pegvaliase treatment in adults with phenylketonuria: Analysis of phase 3 clinical trial data" [Molecular Genetics and Metabolism 141, Issue 3 (2024) 108122]
Fran Rohr, Barbara Burton, Anne Dee, et al.
Human Molecular Genetics
|
August 21, 2003
Mutations in COX10 result in a defect in mitochondrial heme A biosynthesis and account for multiple, early-onset clinical phenotypes associated with isolated COX deficiency
Hana Antonicka, Scot C Leary, Guy-Hellen Guercin, et al.
Molecular Genetics and Metabolism
|
July 26, 2005
Effect of optimal dietary therapy upon visual function in children with long-chain 3-hydroxyacyl CoA dehydrogenase and trifunctional protein deficiency
Melanie B Gillingham, Richard G Weleber, Martha Neuringer, et al.
Molecular Therapy. Nucleic Acids
|
June 19, 2017
Low-Dose Gene Therapy for Murine PKU Using Episomal Naked DNA Vectors Expressing PAH from Its Endogenous Liver Promoter
Hiu Man Grisch-Chan, Andrea Schlegel, Tanja Scherer, et al.
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of 12
Search research articles
Search
Showing results (61-70 of 113) with videos related to
Sort By:
Page
of 12
Molecular Genetics and Metabolism
|
July 22, 2022
Biochemical and behavioural profile of NTBC treated Tyrosinemie type 1 mice
Willem G van Ginkel, Shelley R Winn, Sandra Dudley, et al.
Journal of Child Neurology
|
August 15, 2002
Friedreich's ataxia associated with mitochondrial myopathy: clinicopathologic report
Catherine L Gallagher, Andrew J Waclawik, Brad R Beinlich, et al.
Investigative Ophthalmology & Visual Science
|
September 16, 2024
iPSC-Derived LCHADD Retinal Pigment Epithelial Cells Are Susceptible to Lipid Peroxidation and Rescued by Transfection of a Wildtype AAV-HADHA Vector
Tiffany DeVine, Gabriela Elizondo, Garen Gaston, et al.
Science Translational Medicine
|
June 10, 2021
Therapeutic liver repopulation by transient acetaminophen selection of gene-modified hepatocytes
Anne Vonada, Amita Tiyaboonchai, Sean Nygaard, et al.
Molecular Genetics and Metabolism
|
January 7, 2024
Evaluating change in diet with pegvaliase treatment in adults with phenylketonuria: Analysis of phase 3 clinical trial data
Fran Rohr, Barbara Burton, Anne Dee, et al.
Molecular Genetics and Metabolism
|
February 28, 2026
Management of pegvaliase-related skin concerns: best practice recommendations using a modified Delphi approach
Alvaro Hermida Ameijeiras, Erika Vucko, Cary O Harding, et al.
Molecular Genetics and Metabolism
|
November 26, 2024
Corrigendum to "Evaluating change in diet with pegvaliase treatment in adults with phenylketonuria: Analysis of phase 3 clinical trial data" [Molecular Genetics and Metabolism 141, Issue 3 (2024) 108122]
Fran Rohr, Barbara Burton, Anne Dee, et al.
Human Molecular Genetics
|
August 21, 2003
Mutations in COX10 result in a defect in mitochondrial heme A biosynthesis and account for multiple, early-onset clinical phenotypes associated with isolated COX deficiency
Hana Antonicka, Scot C Leary, Guy-Hellen Guercin, et al.
Molecular Genetics and Metabolism
|
July 26, 2005
Effect of optimal dietary therapy upon visual function in children with long-chain 3-hydroxyacyl CoA dehydrogenase and trifunctional protein deficiency
Melanie B Gillingham, Richard G Weleber, Martha Neuringer, et al.
Molecular Therapy. Nucleic Acids
|
June 19, 2017
Low-Dose Gene Therapy for Murine PKU Using Episomal Naked DNA Vectors Expressing PAH from Its Endogenous Liver Promoter
Hiu Man Grisch-Chan, Andrea Schlegel, Tanja Scherer, et al.
Page
of 12