We are pleased to welcome Professor Krishanu Saha of the University of Wisconsin as our 2019-2020 Rising Star speaker.
Please join us!
Wednesday, November 20
12:00 – 1:00 PM
106 Stanley Hall
“Towards nonviral, in vivo genome editing therapies: new tools and models to facilitate translation “
Krishanu Saha, PhD
Associate Professor
Department of Biomedical Engineering &
Wisconsin Institute for Discovery
Abstract:
Genome editing tools, many developed at UC-Berkeley, have high potential to directly fix or disrupt gene mutations responsible for a wide variety of diseases. Developing safe and effective therapies for in vivo somatic genome editing, however, faces a number of challenges including efficient delivery, manufacturing at scale, and determination of unintended off-target effects in relevant biological systems. We have developed new approaches to inform first-in-human studies that combine patient-derived, induced pluripotent stem cell models with new delivery nanocarriers and computational models. Our results indicate that mutation-specific, CRISPR-Cas9 genome editors can be designed to efficiently target mutant alleles without substantial off-target effects. Further, tailored polymeric nanoparticles can efficiently deliver these editors, simplifying manufacturing by avoiding viral vectors. Finally, computational models for in vivo somatic editing, grounded in empirical data from in vitro models and delivery studies, indicate that injectable off-the-shelf nonviral genome editors can be efficacious. Applications of these tools will be focused on inherited ocular and glycogen storage disorders, and likely generalizable to other rare disorders.
Bio:
Saha studied Chemical Engineering at Cornell University and at UC Berkeley and went on to postdoctoral fellowships at MIT and Harvard. In his dissertation with Professors David Schaffer and Kevin Healy, he worked on experimental and computational analyses of neural stem cell development, as well as the design of new materials for adult stem cell culture. He is the recipient of a 2012 Sage Bionetworks Young Investigator Award, a 2013 BMES Rising Star Award, and a 2014 NSF CAREER Award.
Past Rising Stars
2017-18
Professor Stanley Qi of Stanford University
2016-17
Professor Kim Woodrow of the University of Washington
“Engineering the mucosal microenvironment promotes targeting of particulate and cellular immunotherapies to lymphoid organs”