Showing results (41-50 of 248) with videos related to
Sort By:
Pageof 25
Molecular Therapy. Nucleic Acids|September 17, 2014
A sensitive assay system to test antisense oligonucleotides for splice suppression therapy in the mouse liverLorena Gallego-Villar, Hiu Man Viecelli, Belén Pérez, et al.Molecular Genetics and Metabolism|January 15, 2018
Blood phenylalanine reduction corrects CNS dopamine and serotonin deficiencies and partially improves behavioral performance in adult phenylketonuric miceShelley R Winn, Tanja Scherer, Beat Thöny, et al.Molecular Therapy. Methods & Clinical Development|January 24, 2020
AAV-Mediated CRISPR/Cas9 Gene Editing in Murine PhenylketonuriaDaelyn Y Richards, Shelley R Winn, Sandra Dudley, et al.Journal of Inherited Metabolic Disease|January 15, 2021
Cardiac tissue citric acid cycle intermediates in exercised very long-chain acyl-CoA dehydrogenase-deficient mice fed triheptanoin or medium-chain triglycerideGaren Gaston, Jon A Gangoiti, Shelley Winn, et al.Molecular Therapy. Nucleic Acids|January 22, 2026
Enhancement of therapeutic transgene insertion for treatment of murine phenylketonuriaMichael 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) deficiencyGabriela 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 acidemiaCristy 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 oxidationAnnie 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 neonateCary 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 miceShelley R Winn, Sandra Dudley, Tanja Scherer, et al.Pageof 25