Emilia Caya Blonkenfeld, COL ’25, San Francisco, CA
This summer I had the privilege of continuing my project in the Warren Lab out of CHOP’s Center for Pediatric Comprehensive Bone Marrow Failure. With the help and generosity of Career Services I was able to work through the summer instead of having to pause my work until the following school year. My project concerns itself with a rare inherited bone failure disease called severe congenital neutropenia (SCN).
Patients with SCN are characterized by a chronically low number of white blood cells called neutrophils. This low neutrophil count, or neutropenia, makes them extremely susceptible to infections and the development of blood related cancers. Unfortunately, there is currently only one treatment available for SCN and its nature gives it a low efficiency success and more troublesome leaves patients even more susceptible to developing blood malignancies. Thus, my lab is interested in learning more about the pathway that leads to the differentiation of neutrophils from stem cells with the goal of ultimately developing a way to produce healthy neutrophils for transfusion treatments.
To tackle this really big goal we are performing a knockout screen in which we use CRISPR Cas9 technology to systematically cleave out a target gene to subsequently watch its effect on the ability of the hematopoietic stem cell to differentiate into a neutrophil. For the last 6 months I have been working to build the sub-library of lentiviral plasmid vectors we will use to target these specific genes. Building this library can be broken down into three major steps: designing the genetic sequences that will target our genes of interest, inserting these sequences into bacteria to be cloned, a process called transformation, and finally the purification of this modified bacterial DNA. Each round takes 3 days so needless to say there was a lot of rinse and repeating experimental protocols.
Our final screen will target approximately 150 genes. As of the end of the summer I am only about a third of the way through building the library. Though there is still a lot of work to do each day presents new challenges and new ways to understand the scientific process. I learned a lot this summer about the resilience required in academic science, about how to come back from failure and use mistakes to proactively prevent future ones. I am very excited to return to the lab in the fall and continue to grow as a scientist, student, and researcher.
This is part of a series of posts by recipients of the 2024 Career Services Summer Funding Grant. We’ve asked funding recipients to reflect on their summer experiences and talk about the industries in which they spent their summer. You can read the entire series here
