With the funds just awarded by RSRT, I am now leading a screening effort in the lab of Rudolf Jaenisch at the Whitehead Institute in Cambridge, Massachusetts to identify drugs that can boost KCC2 expression. We are using cutting-edge CRISPR/Cas9 genome editing technology to insert a gene that reports KCC2 expression levels into human Rett Syndrome neurons. Our pilot screen had identified a number of hit compounds that we are now validating. The influx of new funding will allow high-throughput screening to identify more KCC2-enhancing compounds with improved pharmacological properties and validation of hit compounds.
I am excited to be working on a project that has the potential to alleviate suffering in Rett patients and I look forward to keeping RSRT and the Rett community apprised of our work.