Michael Przybilla

20162020

Research output per year

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Research Output

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., Jan 1 2020, (Accepted/In press) In : Gene therapy.

Research output: Contribution to journalArticle

  • Comprehensive behavioral and biochemical outcomes of novel murine models of GM1-gangliosidosis and Morquio syndrome type B

    Przybilla, M. J., Ou, L., Tăbăran, A. F., Jiang, X., Sidhu, R., Kell, P. J., Ory, D. S., O'Sullivan, M. G. & Whitley, C. B., Feb 2019, In : Molecular Genetics and Metabolism. 126, 2, p. 139-150 12 p.

    Research output: Contribution to journalArticle

    Open Access
  • 3 Scopus citations
    Open Access
  • 6 Scopus citations

    ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

    Ou, L., DeKelver, R. C., Rohde, M., Tom, S., Radeke, R., St. Martin, S. J., Santiago, Y., Sproul, S., Przybilla, M. J., Koniar, B. L., Podetz-Pedersen, K. M., Laoharawee, K., Cooksley, R. D., Meyer, K. E., Holmes, M. C., McIvor, R. S., Wechsler, T. & Whitley, C. B., Jan 2 2019, In : Molecular Therapy. 27, 1, p. 178-187 10 p.

    Research output: Contribution to journalArticle

    Open Access
  • 9 Scopus citations
    Open Access
  • 6 Scopus citations