1977 …2020

Research output per year

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Fingerprint The Fingerprint is created by mining the titles and abstracts of the person's research outputs and projects/funding awards to create an index of weighted terms from discipline-specific thesauri.

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Research Output

A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I

Ou, L., Przybilla, M. J., Ahlat, O., Kim, S., Overn, P., Jarnes, J., O'Sullivan, M. G. & Whitley, C. B., Jun 3 2020, In : Molecular Therapy. 28, 6, p. 1442-1454 13 p.

Research output: Contribution to journalArticle

  • 1 Scopus citations

    A novel gene editing system to treat both Tay–Sachs and Sandhoff diseases

    Ou, L., Przybilla, M. J., Tăbăran, A. F., Overn, P., O’Sullivan, M. G., Jiang, X., Sidhu, R., Kell, P. J., Ory, D. S. & Whitley, C. B., May 1 2020, In : Gene therapy. 27, 5, p. 226-236 11 p.

    Research output: Contribution to journalArticle

  • 4 Scopus citations

    Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation

    Polgreen, L. E., Lund, T. C., Braunlin, E., Tolar, J., Miller, B. S., Fung, E., Whitley, C. B., Eisengart, J. B., Northrop, E., Rudser, K., Miller, W. P. & Orchard, P. J., Jan 1 2020, In : Pediatric Research. 87, 1, p. 104-111 8 p.

    Research output: Contribution to journalArticle

    Open Access
  • 2 Scopus citations
    1 Scopus citations

    The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII

    Wang, R. Y., da Silva Franco, J. F., López-Valdez, J., Martins, E., Sutton, V. R., Whitley, C. B., Zhang, L., Cimms, T., Marsden, D., Jurecka, A. & Harmatz, P., Mar 2020, In : Molecular Genetics and Metabolism. 129, 3, p. 219-227 9 p.

    Research output: Contribution to journalArticle

    Open Access