HIGHLIGHTS
- who: Jon Cooper from the DepartmentWashington University St Louis, MO, USA have published the article: Nelvagal JCI Second Resubmission 8-19 CLEAN With Figs, in the Journal: Neurology,
- what: The authors tested this repeated dosing strategy first in Cln1-/- mice, a well-established and characterized model recapitulating most aspects of classical CLN1 disease , using a previously defined dose and frequency based on enzyme half-life . In the current study, brain and spinal cord tissue were collected 24 h after the final infusion at six months. While this study is focused on a single rare disorder . . .
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