Le, Steven Q; Kan, Shih-Hsin; Clarke, Don et al. (2018) A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I. Mol Ther Methods Clin Dev 8:42-51
|
Clarke, Don; Pearse, Yewande; Kan, Shih-Hsin et al. (2018) Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice. Mol Ther Methods Clin Dev 10:113-127
|
Angarita, Stephanie A K; Truong, Brian; Khoja, Suhail et al. (2018) Human hepatocyte transplantation corrects the inherited metabolic liver disorder arginase deficiency in mice. Mol Genet Metab 124:114-123
|
Gallant, N M; Leydiker, K; Wilnai, Y et al. (2017) Biochemical characteristics of newborns with carnitine transporter defect identified by newborn screening in California. Mol Genet Metab 122:76-84
|
Marques, Felipe; Tenney, Jessica; Duran, Ivan et al. (2016) Altered mRNA Splicing, Chondrocyte Gene Expression and Abnormal Skeletal Development due to SF3B4 Mutations in Rodriguez Acrofacial Dysostosis. PLoS Genet 12:e1006307
|
Kan, Shih-Hsin; Le, Steven Q; Bui, Quang D et al. (2016) Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice. Behav Brain Res 312:265-71
|
Wang, Jessica Jen-Chu; Rau, Christoph; Avetisyan, Rozeta et al. (2016) Genetic Dissection of Cardiac Remodeling in an Isoproterenol-Induced Heart Failure Mouse Model. PLoS Genet 12:e1006038
|
Lee, Patrick C; Truong, Brian; Vega-Crespo, Agustin et al. (2016) Restoring Ureagenesis in Hepatocytes by CRISPR/Cas9-mediated Genomic Addition to Arginase-deficient Induced Pluripotent Stem Cells. Mol Ther Nucleic Acids 5:e394
|
Tai, Denise S; Hu, Chuhong; Kim, Elizabeth H et al. (2015) Augmentation of transgene-encoded protein after neonatal injection of adeno-associated virus improves hepatic copy number without immune responses. Pediatr Res 78:239-246
|
Rickabaugh, Tammy M; Baxter, Ruth M; Sehl, Mary et al. (2015) Acceleration of age-associated methylation patterns in HIV-1-infected adults. PLoS One 10:e0119201
|
Showing the most recent 10 out of 130 publications