Mariah J. Lelos

This study demonstrates that a single-AAV vector delivering the SpCas9 nickase and sgRNA achieves greater CAG/CTG repeat contraction in a Huntington's disease mouse model compared to a dual-vector system.

The paper uses MRI and histological analyses to show axonal and myelin changes in a Huntington's Disease mouse model, identifying FR as a potential MRI biomarker for WM microstructure changes.

This study shows that hPSC-derived medium spiny neurons (hPSC-MSNs) do not fully replicate the epigenetic and transcriptional features of authentic fetal MSNs, highlighting limitations in their fidelity.

The paper demonstrates that CRISPR-Cas9 nickase can contract CAG/CTG repeats in patient-derived cells and improve molecular and behavioral phenotypes in Huntington's disease.

This study shows that acyl-ghrelin reduces dopamine neuron loss and motor deficits in a rat model of Parkinson's disease.

This study shows that functional recovery from dopamine neuron grafts depends on neurite outgrowth, not graft size or neuron content, and identifies gene expression patterns linked to graft efficacy.