DINNER SHORT COURSE: TUESDAY, AUGUST 31, 6:30 - 9:00 PM

CRISPR/Cas9 Applications in Immunotherapy

Detailed Agenda

6:30 Dinner Buffet

6:45 CRISPR Editing Human T cells: Knock-outs, Knock-ins and Screening Approaches

Kathrin Schumann, Ph.D., Fellow, Marson Lab, University of Californa San Francisco

Genetic manipulation of human T cells has been largely impossible until recently, but advances in genome engineering offer new opportunities. We developed a robust CRISPR/Cas9 technology based on Cas9 ribonucleoproteins (Cas9 RNPs) that enables both “knock-out” and “knock-in” genome editing in primary human T cells. We expanded this technique to genes associate with cancer, HIV and autoimmune diseases and developed a high-throughput platform to perform screens in human T cells.

7:45 Genome Editing: Delivery and Clinical Trials

Hao Yin, Ph.D., Research Scientist, The David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology

With the recent development of CRISPR technology, it is becoming increasingly easy to engineer the genome. Genome-editing systems based on CRISPR, as well as transcription activator-like effector nucleases (TALENs) and zinc-finger nucleases (ZFNs), are becoming valuable tools for biomedical research, drug discovery and development, and even gene therapy. However, for each of these systems to effectively enter cells of interest and perform their function, efficient and safe delivery technologies are needed. We will discuss the principles of biomacromolecule delivery and gene editing, examines recent advances and challenges in non-viral and viral delivery methods, and highlights the status of related clinical trials.

8:45 Extended Q&A with Course Instructors

9:00 Close of Course

Instructor Biographies

Kathrin Schumann, Ph.D., Fellow, Marson Lab, University of Californa San Francisco

Kathrin Schumann studied Biochemistry in Tübingen and Munich, Germany. During her diploma and PhD thesis in Michael Sixt’s lab at the Max Planck Institute of Biochemistry near Munich she analyzed how the presenting mode of chemokines influences dendritic cell migration. After her PhD, she decided to gain experience in translational aspects of research and drug development. She joined the Novartis Postdoc Program in Basel, Switzerland, working within the autoimmune disease area. In March 2014 she joint the labs of Alexander Marson and Jeffrey Bluestone to establish genome engineering in human primary T cells for experimental and cell therapeutic applications.

Hao Yin, Ph.D., Research Scientist, The David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology

Biography Unavailable