Spotlight on Gene Therapy: finding a Cure

Our lab published "Therapeutic rAAVrh.10-mediated SOD1 silencing in adult SOD1G93A mice and non-human primates". We are also making the cover of the January issue of Human Gene Therapy! Full text available here.

Our lab in collaboration with the lab of Dr. Brown published "Human C9ORF72 Hexanucleotide Expansion Reproduces RNA Foci and Dipeptide Repeat Proteins but Not Neurodegeneration in BAC Transgenic Mice".