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EB2017: The CRISPR-Cas9 Revolution in Pharmacology

April 26, 2017
By Tamara Escajadillo

Monday morning kicked into high gear with a wide variety of sessions to choose from, including one on the way CRISPR-Cas9 has revolutionized the field of pharmacology. Since before the session even began, the meeting room was completely packed, and even standing room in the back was limited, to say the least. After a brief introduction by chair Karen Tonsfeldt, Dr. Charles Gersbach from Duke University kicked off the session by talking about how, in this new phase of genomic engineering, we are capable of not only using genome editing therapeutically, but as a way to develop new tools in research as well. His research focuses mainly on a genetic disease called Duchenne muscular dystrophy, which affects approximately one in every 5000 males. Since the full-length dystrophin gene responsible for the pathology is too large to package for delivery using traditional methods, the CRISPR-Cas9 system has emerged as a way to restore dystrophin protein expression and function that can potentially be done directly into patient muscle cells in the future. Further talks included the generation of Knock-out and Knock-in primary T cells using Cas9 ribonucleases by Dr. Kathrin Schumann from UCSF, CRISPRing out opiate reward by manipulation of the neurokinin system by Dr. Alexander Sandweiss from the University of Arizona, and CRISPR-Cas9 mediated genome editing of murine pulmonary circulation by Dr. Glenn Marsboom from the University of Illinois at Chicago.

Aside from the great talks given by the speakers, one of the added benefits of attending this session was the ability for the audience to participate in a discussion with the speakers during the short stretch break. In the realm of clinical applications, topics brought up by audience members included the possibility of using CRISPR to make targeted personalized cancer therapies, and the hope of being able to use this system to “CRISPR-out” HIV virus from patient cells. The issue of how to restrict the Cas9 expression in order to decrease off target effects emerged as well, and the panelists agreed that although there is tropism for certain tissues, the system is not perfect (yet) and there is the possibility of triggering immune responses, although in their research these immune responses have not been robust enough to clear away the newly transduced cells. Given the wide breadth of advances that have emerged since the CRISPR-Cas9 methods for gene editing have come into play, we can all expect even more great results from its utilization in future studies!

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Last Updated: September 6, 2017

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