February 21-23 2017

Boston, USA


Workshop A
Thursday February 23rd, 2017

09.00 - 12.00
Non-Traditional Organism Models for Human Disease
Workshop Leader: Manush Mohammed, Research Instructor, Developmental Biology , University of Pittsburgh Medical School Workshop Leader: Jeffrey Essner, Associate Professor, Department of Genetics, Development & Cell Biology, Iowa State University

Increasing our understanding of the cellular mechanisms responsible for laterality defects using zebrafish model is critical for the development of therapeutic approaches and devising early intervention strategies in humans during development.

Key areas of discussion include:

  • Advantages of using zebrafish as a disease model for humans
  • Latest updates on challenges and opportunities of using zebrafish genetics
  • Discuss alternative approaches to overcome these challenges
  • Using TALEN and CRISPR-Cas9 systems robustly activated MMEJ in zebrafish in vivo
  • To present key parameters for determining MMEJ sequences to enable genome engineers to utilize MMEJ for many new exciting applications such as highly efficient knock-ins and precise, tissue-specific gene knock-out in animals, human cells in vitro, and for human gene therapy

Manush Mohammed, Research Instructor, Developmental Biology , University of Pittsburgh Medical School

manush-mohammed-photo

Jeffrey Essner, Associate Professor, Department of Genetics, Development & Cell Biology, Iowa State University

Speaker

Dr. Essner received his Ph.D. from the University of Minnesota with Dr. Perry Hackett, where he began using the zebrafish model system to understand the cellular roles of proto-oncogenes. Prior to joining the faculty of the Genetics, Development and Cell Biology Department at Iowa State University in 2005, Dr. Essner was the Scientific Director at Discovery Genomics, Inc., a biotech company that uses zebrafish for high-throughput analysis of gene function. At Iowa State University, Dr. Essner has developed methods for targeted mutagenesis using TALENs and CRISPR/Cas9 for modeling human disease in zebrafish with a focus on angiogenesis and cancer progression.

Workshop B
Thursday February 23rd, 2017

13.00 - 16.00
Taking CRISPR/Cas9 to the Clinic: Learnings from Gene Therapy Applications of Gene Editing
Workshop Leader: Christian Mueller, Associate Professor, Pediatrics & Gene Therapy Center , University of Massachusetts Medical School

Given the enormous impact CRISPR gene editing is set to have in developing gene therapies, the industry are advancing and adapting the technology to get it ready for clinical use.

Questions that will be addressed include:

  • How are the latest industry developments using CRISPR precision gene editing for gene therapies?
  • What clinical lessons learnt can we take away from other gene editing tools when applying CRISPR in the clinic?
  • What are the safety considerations and how is the industry working to make CRISPR technology safe for therapeutic use?

Christian Mueller, Associate Professor, Pediatrics & Gene Therapy Center , University of Massachusetts Medical School

chris-mueller