SC12: Introduction to Gene Editing
Monday September 19 | 3:30-6:30 pm


This course will cover the basics of gene editing, the terminologies and the techniques, the applications, their strengths and limitations. The course will discuss the differences between the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats)/Cas, Transcription Activator-like Effector Nucleases (TALENs), Zinc Finger Nucleases (ZFNs) and other gene editing systems. It will introduce the concepts of CRISPRi, CRISPRa, when and where they can be used. Alternatives of Cas9, basics of guide RNA design and mechanism of action, will all be discussed in an informal, interactive setting. There will be plenty of time allocated to open discussion, sharing of ideas and exchange of best practices, as people are updated on the scientific and technical progress being made in the field, as well as a quick overview of the regulatory landscape.

3:30 Welcome and Speaker Introductions

3:45 Introduction to Gene Editing in Cells and In vivo

Stephanie Mohr, PhD, Lecturer, Genetics & Director, Drosophila RNAi Screening Center at Harvard Medical School

  • Introduction to gene editing, from protein-based systems to the CRISPR protein-RNA system
  • Expanding the CRISPR toolbox: CRISPR-inhibition and activation
  • In vivo applications of CRISPR technologies

4:45 Coffee Break

5:05 Bioinformatics for CRISPRs

Claire Yanhui Hu, Ph.D, Senior Bioinformatician, Drosophila RNAi Screening Center, Department of Genetics, Harvard Medical School

  • Bioinformatics analysis of low-and high-throughput CRISPR data
  • Considerations when designing CRISPR gRNAs for different purposes, eg. knock-out, knock-in or activation, or for different organisms
  • Case-study in gRNA selection for Drosophila and tips on selecting the right tool for your system

5:45 Regulatory Landscape for CRISPR-Based Research

Paul Enríquez, J.D., LL.M., Ph.D. Candidate, Structural and Molecular Biochemistry, North Carolina State University

  • Regulatory overview of CRISPR-based research.
  • Past lessons from stem cell research and gene therapy.
  • Recommendations for CRISPR scientists.

BIOS

Paul Enríquez, J.D., LL.M., Ph.D. Candidate, Structural and Molecular Biochemistry, North Carolina State University

Paul Enríquez is in the Department of Structural and Molecular Biochemistry at North Carolina State University. He holds undergraduate degrees in Biochemistry, Business Management, and Genetics; graduate degrees in Law (J.D.), International & Comparative Law (LL.M.); and is currently conducting research for his Ph.D. He is licensed to practice law in the State of North Carolina* and has also passed the New York Bar Exam. His work has been featured in both legal and scientific journals. In his time outside the lab, Paul writes about the intersection of law and science and is actively researching the legal implications raised by CRISPR-based scientific research.

Stephanie_MohrStephanie Mohr, PhD, Lecturer, Genetics & Director, Drosophila RNAi Screening Center at Harvard Medical School

Stephanie Mohr is the Director of the Drosophila RNAi Screening Center (DRSC) at Harvard Medical School, which supports functional genomics screens and analyses by the Drosophila community, and Director of the Dana Farber/Harvard Cancer Center Functional Genomics Core. She also uses the DRSC platform to conduct original research. Stephanie received her BA from Wesleyan University and Ph.D. from the University of Colorado, Boulder. Prior to joining the DRSC, she was a postdoc with Prof. William M. Gelbart at Harvard University and scientific liaison in the laboratory of Dr. Josh LaBaer at Harvard Medical School.

Claire_HuClaire Yanhui Hu, Ph.D, Senior Bioinformatician, Drosophila RNAi Screening Center, Department of Genetics, Harvard Medical School

Yanhui (Claire) Hu is a bioinformatician with more than 10 years of experience in mammalian, Drosophila and other model systems. As bioinformatician at the Harvard Institute of Proteomics (2000-2010), Dr. Hu developed two text-mining tools, MedGene and BioGene, which have been licensed by pharmaceutical companies as well as being used by the academic research community to identify genes associated with human diseases and biological/chemical themes from PubMed literature., and made many other contributions to research and community resource projects. Since 2010, Dr Hu has been the senior bioinformatician at the Drosophila RNAi Screening Center (DRSC), working closely with researchers, programmers and others to accomplish common goals in reagent design, reagent identification, data analysis and more. At the DRSC Dr. Hu has developed an integrative tool for ortholog predictions among major model organisms, DIOPT, as well as the related human disease gene ortholog prediction tool, DIOPT-DIST, and applied these tools in various ways, including for disease-focused studies in Drosophila. She has also developed or otherwise contributed to several other projects in the areas of reagent design (qPCR primers, CRISPR gRNAs), reagent identification (UP-TORR), project design (HuDis-TRIP), and data analysis (COMPLEAT and SignedPPI). Each of the online resources Dr. Hu developed is freely available online at www.flyrnai.org.