Proteomic analysis of muccopolysaccharidosis I mouse brain with two-dimensional polyacrylamide gel electrophoresis

Por Ou, Li, Przybilla, Michael J, Whitley, Chester B
Publicado no Mol Genet Metab (2016)

Phenotype prediction for mucopolysaccharidosis type I by in silico analysis

Por Ou, Li, Przybilla, Michael J., Whitley, Chester B.
Publicado no Orphanet J Rare Dis (2017)

SAAMP 2.0: an algorithm to predict genotype-phenotype correlation of lysosomal storage diseases

Por Ou, Li, Przybilla, Michael J, Whitley, Chester B
Publicado no Clin Genet (2018)

Metabolomics profiling reveals profound metabolic impairments in mice and patients with Sandhoff disease

Por Ou, Li, Przybilla, Michael J, Whitley, Chester B
Publicado no Mol Genet Metab (2018)

RTB lectin-mediated delivery of lysosomal α-L-iduronidase mitigates disease manifestations systemically including the central nervous system

Por Ou, Li, Przybilla, Michael J, Koniar, Brenda, Whitley, Chester B
Publicado no Mol Genet Metab (2017)

Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I

Por Ou, Li, Przybilla, Michael J., Koniar, Brenda L., Whitley, Chester B.
Publicado no Mol Genet Metab Rep (2016)

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

Por Ou, Li, DeKelver, Russell C., Rohde, Michelle, Tom, Susan, Radeke, Robert, St. Martin, Susan J., Santiago, Yolanda, Sproul, Scott, Przybilla, Michael J., Koniar, Brenda L., Podetz-Pedersen, Kelly M., Laoharawee, Kanut, Cooksley, Renee D., Meyer, Kathleen E., Holmes, Michael C., McIvor, R. Scott, Wechsler, Thomas, Whitley, Chester B.
Publicado no Mol Ther (2019)