Christopher M. Treleaven

TL;DR: Increasing glucocerebrosidase activity in the CNS represents a potential therapeutic strategy for GBA1-related and non-GBA1–associated synucleinopathies, including PD.

TL;DR: Testing an experimental drug that inhibits the production of glucocerebrosides suggests that producing fewer lipids may counteract the challenge of clearing them from cells, and supports the contention that prolonged antagonism of GCS in the CNS can affect α-synuclein processing and improve behavioral outcomes.

TL;DR: It is demonstrated for the first time that IGF-1 attenuates the pathological activity of non-neuronal cells that contribute to disease progression and attenuates glial cell-mediated release of tumor necrosis factor-alpha (TNF-alpha) and nitric oxide (NO).

TL;DR: This study examines the merits of administering a recombinant adeno-associated viral vector designed to deliver small interfering RNA (siRNA) that targets the degradation of the Htt transcript to lower Htt levels and correct the behavioral, biochemical, and neuropathological deficits shown to be associated with the YAC128 mouse model of HD.

TL;DR: It is shown that adeno-associated virus serotype 4 (AAV4)-mediated expression of insulin-like growth factor-1 (IGF-1) or vascular endothelial growth factor (VEGF)-165 in the cellular components of the ventricular system leads to trophic factor delivery throughout the CNS, delayed motor decline and a significant extension of survival in SOD1(G93A) transgenic mice.