Emily M. Rocha

TL;DR: It is demonstrated for the first time that an age‐dependent reduction in glucocerebrosidase activity may lower the threshold for developing Parkinson's disease.

TL;DR: The neuroprotective effects of increasing GCase against dopaminergic neuron degeneration are demonstrated, for the first time, and support the development of therapeutics targeting GCase or other lysosomal genes to improve neuronal handling of α-synuclein.

TL;DR: It is demonstrated that systemic reduction of GCase activity using chemical inhibition, leads to neuropathological changes in the brain reminiscent of α-synucleinopathy, which is linked to sporadic PD and normal aging.

TL;DR: It is shown that the hSYN1 promoter can be used with rAAV9 to drive robust neuron-specific transgene expression throughout the nervous system, and GFP expression was identified in several neuronal sub-types, including nigral tyrosine hydroxylase (TH)-positive dopaminergic cells, striatal dopamine- and cAMP-regulated neuronal phosphoprotein (DARPP-32)-positive neurons, and choline acetyltransferase (ChAT)-

TL;DR: Data from this study indicate that the heterozygous Q175 KI mouse represents a realistic model for Huntington's disease and also provides new insights into the specific and progressive synaptic, cytoskeletal and axonal transport protein abnormalities that may accompany the disease.