CB 399: The CRISPR-Cas9 System and the Future of Genome Editing Fall 2014

The CRISPR-Cas9 System and the Future of Genome Editing

Nanocourse Director: Dr. Fred Winston
Curriculum Fellow: Emily Gleason, Emily_Gleason@hms.harvard.edu 


  • J. Keith Joung, MD, PhD, Associate Chief of Pathology for Research and Associate Professor of Pathology, Massachusetts General Hospital 
  • Kiran Musunuru, MD, PhD, MPH, Assistant Professor, Department of Stem Cell and Regenerative Biology, Harvard University
  • Stephanie Mohr, PhD, Director of the Drosophila RNAi Screening Center, Co-Director of the Genome Engineering Production Group, and Coordinator of the DF/HCC Collaborative RNAi Core, Harvard Medical School

Clustered regularly interspaced short palindromic repeats (CRISPRs) and their associated endonuclease, Cas9, are an important part of the immune systems of many prokaryotes. CRISPR-Cas systems act as RNA-directed endonucleases that target nucleic acids in a sequence-specific manner. Importantly, CRISPR-Cas9 systems have been shown to be highly adaptable. In 2012, it was demonstrated that Cas9 could be directed to different target sites simply by changing the sequence of an associated RNA, leading to speculation that the system could be used as a genome-editing tool. This discovery kicked off the rapid development and refinement of CRISPR-based genome-editing tools as well advances in using this technology both to further basic research and to develop potential therapies for a number of human diseases. In this course, we will provide an introduction to genome editing and the development of CRISPR-Cas systems. We will also highlight some potential applications of CRISPR-Cas9 towards both basic research and human therapeutics. Finally, we will provide an overview of some of the resources available at Harvard for researchers interested in using CRISPR-Cas9 genome editing to further their projects.  

Schedule: (First session is open to public, second is for registered students only)

First Session: Thursday, November 6th 9:30 AM – 1 PM
Location: New Research Building, Rm. 1031
Second Session: Thursday, November 13th 10 AM – 12:30 PM
Location: TMEC, Rm L-008 

For the second session of the course, we will be holding a ‘grant panel’. Students will consider how they might apply the CRISPR-Cas9 system to their own research projects or interests. Students will write a 1-page single-spaced proposal (maximum 500 words) describing a project applying some of the concepts and tools discussed in the first session.

Proposals should contain the following elements:

  • A title
  • A brief introductory paragraph that describes the question you are asking, your hypothesis and introduces the model system you are working with.
  • 2 or 3 specific aims
  • A synopsis of the experiments you plan to conduct to address those aims.
  • Typically, specific aims pages do not contain figures.

Additionally, proposals are expected to describe experiments that go beyond simply using CRISPR-Cas9 to knock out a gene either by proposing supporting experiments or by exploring issues related to the creation of the knockout model such as off-target effects. 

Proposals must be e-mailed to Emily (emily_gleason@hms.harvard.edu) no later than 3pm on Tuesday, November 11th and be submitted in either .doc, .docx, or .pdf formats with your last name included in the file name. Emily will compile the proposals and email them to the class and to the teaching staff. You are expected to read your classmates’ proposals and come to class prepared to discuss them. 

DROP DEADLINE: Thursday, October 30, 2014