Seelos' Single Dose Of SLS-004 Shows Reduction In Parkinson's Associated mRNA, Protein Expression

Seelos Therapeutics Inc SEEL has announced positive in vivo data demonstrating down-regulation of SNCA mRNA and protein expression from a study of SLS-004.
The SNCA gene has been implicated as a highly significant genetic risk factor for Parkinson's Disease (PD).
In addition, accumulating evidence has suggested that elevated alpha-synuclein levels (α-synuclein) are causative in the pathogenesis of PD.
The cohort administrated with LV vector carrying one key repressor, KRAB-MeCp2, and gRNA targeted the mouse SNCA intron 1, produced a 27% reduction on SNCA mRNA and a 40% reduction in SNCA protein expression in the substantia nigra of the injected left hemisphere versus the right hemisphere.
The cohort administrated with LV vector carrying a different effector, DNMT3A, and the same gRNA showed a more subtle effect on both SNCA-mRNA and protein levels (an average of nearly 10% and 20% reduction, left vs. right, respectively).
SLS-004 is a novel epigenome-editing approach to modulate the expression of SNCA gene mediated by modification of DNA-methylation.
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