Fingerprint Dive into the research topics where Pediatrics - Genetics and Metabolism is active. These topic labels come from the works of this organization's members. Together they form a unique fingerprint.

  • Network Recent external collaboration on country level. Dive into details by clicking on the dots.

    Projects

    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

    Activities

    • 1 Types of Award - Prize (including medals and awards)

    Doctoral Dissertation Fellowship

    Li Ou (Recipient)

    Sep 2015Aug 2016

    Activity: Other activity typesTypes of Award - Prize (including medals and awards)

    Prizes

    American Society of Gene and Cell Therapy Travel Award

    Ou, Li (Recipient), May 2016

    Prize: Prize (including medals and awards)

    Baosteel Fellowship for Excellent Students

    Ou, Li (Recipient), 2009

    Prize: Prize (including medals and awards)

  • NIH Lysosomal Disease Network Fellow

    Ou, Li (Recipient), 2015

    Prize: Prize (including medals and awards)