Congrats to all involved in obtaining funding from the Maryland Stem Cell Research Fund. Our lab, in collaboration with the Ivy Dick lab, was awarded a 2-year MSCRF Launch grant titled “iPSC Platforms to Develop Genome Restoration Strategies Using Cas9-RC“.

The first study, led by the Mackis Lab at Harvard University and featured in Cell Reports, develops an approach for delivering genes, including Cas9, to select neural populations by in utero electroporation. This method achieves internally-controlled mosaicism that facilitates phenotype discovery in the developing brain.

The second paper, from the Suk Lab at UMSOM and published in ACS Nano, demonstrates our ability to target Cas9 mRNA to select foci in the adult brain. This was achieved through systemic delivery using lipid nanoparticles, coupled with localized targeting by microbubble-mediated focused ultrasound. This non-viral strategy represents a significant advance in treating adult brain disorders by enabling focal genome editing directly in the adult brain.

Together, these studies represent substantial progress in non-viral CRISPR delivery methods for both developing and adult brains. By expanding the in vivo CRISPR toolkit, we are laying the groundwork for the development of neural somatic cell genome editing as future genomic therapeutics.

Congratulations Luciano and Gijung!