Want to deliver gene therapies broadly, but activate them only in diseased neurons? TDP-REG "hacks" TDP cryptic mis-splicing to drive expression only WHEN and WHERE needed! Less toxicity, wider therapeutic window, new options for sporadic ALS therapies. biorxiv.org/content/10.110…

Our preprint is out! Connor Maltby used CANVAS patient iNeurons to interrogate how AAGGG repeats in RFC1 cause neurodegeneration. Findings suggest repeat removal but not RFC1 replacement corrects phenotypes -pointing way to a potential therapy. #Ataxia biorxiv.org/content/10.110…

Early registration deadline for #RNASRC will be here before you know it! Florence Besse, Eric T Wang, @gavislab, and Stavroula Mili NIH have an amazing agenda planned for the best and newest RNA research. Register by May 26 for RNASRC and save your space: hubs.ly/Q02wRnt90

#RepeatExpansionDisorders are up to three times more common than current estimates. Underdiagnosis or reduced penetrance? by our wonderful dream team kibanez Matteo Zanovello Chris Clarkson + Bharati Jadhav & Andy Sharp nature.com/articles/s4159…

Our paper on cryptic splicing-controlled protein expression, which we hope will inspire safer and more adventurous gene therapy approaches for ALS and FTD, has been published in Science Magazine science.org/doi/10.1126/sc…