Webinar: Re-Writing the Genome

March 12, 2018 | Posted by Team in Webinar |

Combining Gene Editing and iPSC Technologies
for Preclinical Discovery and Translational Research

Watch our webinar on-demand to hear Ruby Chen-Tsai, Ph.D., CSO of Applied StemCell, discuss how her team is using genome engineering and iPSC technologies to advance disease modeling, lead compound discovery, and bioproduction.


You will learn about:

  • Using genome engineering technologies for the generation of preclinical research models
  • How genome editing technologies can be applied to the development of cell and gene therapies
  • Application of gene editing to bioproduction
  • CRISPR-mediated gene editing for the development of nucleic acid reference standards

About Our Speaker

Ruby Yanru Chen-Tsai, Ph.D. has worked on the development and modification of stem cells and pre-clinical models for over 25 years. Prior to Applied StemCell, Dr. Chen-Tsai was at Stanford University, where she served as Director of the Transgenic Research Center and Associate Director of the Stanford Cancer Institute. Dr. Chen-Tsai is a co-inventor of the TARGATT™ integrase technology and the “DICE” platform for site-specific gene insertion. Her current research focuses on pre-clinical model development, as well as gene- and stem cell-based therapies using genome editing technologies including CRISPR/Cas9 and TARGATT™ integrase.

Science Exchange Stories: Ethan Perlstein, Perlstein Lab

September 25, 2014 | Posted by Tess Mayall in Scientist Profile |

Perlstein Lab Cropped

I recently spoke with our user Ethan Perlstein, whose one-of-a-kind independent lab is flipping traditional drug discovery on its head. Check out how he is changing the paradigm of traditional research, pharmacology, and more below.

Q: What is the focus of the Perlstein Lab?

Ethan: The Perlstein Lab is focused on personalized orphan drug discovery. We take a two-pronged approach. We first create a primordial disease model for a given patients’ mutation; that involves taking a change in the DNA that you see in the disease and putting it into the model organisms.

We use yeast, worms, flies, and fish that have ancestral versions of that gene. We can use those models to do drug discovery, and we can validate the hits that we get in patient derived cells of the same genotype. So it’s a closed system where everything is personalized from the outset.

Q: How did it come into existence? What was the progression from your very first crowdfunding experience to starting your own lab?

Ethan: The science behind it has been incubating a long time, since I was in grad school, so it’s been a ten-year process. Screening using a model organism is something I did in grad school, so it’s existed for awhile. As a post-doc, I took some of those scientific concepts and drilled down deeper, so that put me in a good position to have a scientific foundation.

I spent the next 18 months leaving academia and navigating the business side. Last fall, I put together a business plan, had it reviewed by business people, improved my plan, and by the end of 2014 I began fundraising.

The team started to come together in early April. The lab started to come together in terms of equipment and structure in mid-April. And now we have a fully functional lab that has yeast, worms, and flies, and it’s off to the races. Read the rest of this entry »

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