People

While we have long known the location of disease-causing mutations in the genome, the discovery of CRISPR finally gave us the ability to correct these typos back to what they should be in healthy patients. While this effort has yielded novel therapies in the clinic, in my own lab I want to look beyond simply correcting DNA typos and instead use genome editing to introduce novel functions into cells for therapeutic purposes.
Examples include:
-Engineering red blood cells to deliver novel protein payloads

My Research Interests are divided into 3 areas of focus:

1. Supportive Care (especially Invasive Fungal Infections) following Hematopoietic Stem Cell Transplantation
2. Transplantation for Severe Combined Immunodeficiency
3. Transplantation for Rare Leukemias (JMML and APL)

Our lab's goal is to understand the genetic circuits that control human immune cell function in health and disease. We have begun to identify how genetic risk variants for autoimmune diseases disrupt immune cell circuits (Farh and Marson et al., Nature 2015; Simeonov et al., Nature, 2017), and how pathogenic circuits may be targeted with novel therapeutics (Xiao et al., Immunity 2014). My lab has developed new tools for efficient CRISPR genome engineering in primary human T cells (Schumann et al., PNAS 2015; Roth et al., Nature 2018; Nguyen et al. Nature Biotech 2020).