Complex III deficiency due to an in-frame MT-CYB deletion presenting as ketotic hypoglycemia and lactic acidosis

by Mari Mori, Jennifer Goldstein, Sarah P. Young, Edward H. Bossen, John Shoffner, Dwight D. Koeberl
Published 2015-09-01

Assessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease

by Gensheng Wang, Sarah P Young, Deeksha Bali, Julie Hutt, Songtao Li, Janet Benson, Dwight D Koeberl
Published 2014-01-01

Corrigendum to âAssessment of toxicity and biodistribution of recombinant AAV8 vectorâmediated immunomodulatory gene therapy in mice with Pompe diseaseâ

by Gensheng Wang, Sarah P Young, Deeksha Bali, Julie Hutt, Songtao Li, Janet Benson, Dwight D Koeberl
Published 2015-01-01

Diurnal variability of glucose tetrasaccharide (Glc4) excretion in patients with glycogen storage disease type III

by Sarah P. Young, Aleena Khan, Ela Stefanescu, Andrea M. Seifts, Ghada Hijazi, Stephanie Austin, Priya S. Kishnani
Published 2021-03-01

Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia

by Hye-Ri Kang, Lauren Waskowicz, Andrea M. Seifts, Dustin J. Landau, Sarah P. Young, Dwight D. Koeberl
Published 2019-06-01

Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease

by Sang-oh Han, Songtao Li, Angela McCall, Benjamin Arnson, Jeffrey I. Everitt, Haoyue Zhang, Sarah P. Young, Mai K. ElMallah, Dwight D. Koeberl
Published 2020-06-01