Browsing by Author "Przybilla, Michael J"

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    Optimal Stoichiometry of the PS Gene-editing System
    (2023-11) Saveraid, Hanna G; Przybilla, Michael J; Ou, Li; Whitley, Chester B
    Introduction: The PS Gene-editing System has been shown to increase IDUA expression and improve therapeutic outcomes in murine models of Hurler Syndrome. However, the optimal ratio of bipartite AAV vectors in the system is unknown. Results: Mice (+/- or -/- for IDUA) treated with a 1:6 ratio of AAV1:AAV2 in the PSG System had the highest IDUA activity in plasma and liver, followed by 1:13 and then 1:27 ratio. All treated mice had higher enzyme levels than control mice. Conclusion: This work demonstrates that a 1:6 ratio is optimal; however, all ratios resulted in supraphysiological levels of IDUA activity. More research is needed to determine if other ratios (e.g., 1:1), are more effective.