BME Seminar: New platform technologies for targeted in vivo gene editing
Monday, February 16, 2015
11:45 am - 12:45 pm
Hudson Hall 208
James Dahlman, PhD
Once viewed as a passive link between DNA and protein, RNAs are now known to actively modify gene expression. siRNAs can temporarily reduce the expression of any protein coding gene, miRNAs can suppress the translation of many genes simultaneously, and CRISPR-Cas9 systems enable scientists to make targeted and permanent changes in genomic DNA. Since RNAs specifically and precisely regulate genes, they could revolutionize the way we treat disease. However, the full scientific and clinical potential of RNAs is limited by our inability to deliver them to the right cells in vivo. In vivo delivery is challenging; RNAs must be (a) protected from degradation, (b) delivered to the correct tissue, and (c) ferried into the right cell, without setting off an immune response. While nanoparticles have delivered RNAs to the liver, delivery to other cells has remained difficult. In this presentation, I will describe new tools for in vivo RNA delivery and gene editing. One tool, a nanoparticle named 7C1, has delivered RNA to the heart and lung at low doses, has delivered five different RNAs concurrently in vivo, is being considered for clinical trials, and has been used by ten labs across the United States to study genes associated with disease. Unlike many other nanoparticles, 7C1 does not transfect hepatocytes in vivo.