- Key considerations when designing a gene editing experiment
- Takara Bio’s footprint-free gene editing workflow
- Case study: insertion of a tyrosinemia-related SNP
Using hiPS gene editing to create a tyrosinemia disease model
CRISPR/Cas9 technology enables precise, footprint-free gene editing. By combining CRISPR/Cas9 with human pluripotent stem cells, we can generate disease models that advance our understanding of disease mechanisms and guide development of novel therapeutics. However, creating these models can be quite difficult. Editing genes while concurrently establishing clonal populations can be both inefficient and time consuming.
In this presentation, Dr. Montse Morell talks about the challenges of creating disease models from hiPSCs. She shares a streamlined method for generating iPSC-derived hepatocytes to model tyrosinemia.
Dr. Montse Morell received her bachelor’s degree with honors in Chemistry from Universitat Autonoma de Barcelona in 2003. In 2008, she completed her PhD in Biotechnology at the same university. Her thesis work focused on the development of protein reporters to study in vivo protein interactions and aggregation. Afterward, she performed her postdoctoral work at Stanford University, where she developed fluorescent probes to detect the activity of metaloproteases in vivo. During her 8 years at Takara Bio USA, Inc., she played an instrumental role in the development of products related to genome engineering using CRISPR/Cas9.
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