1. PhD training in the Lek lab at Yale (2018-2023)
My PhD projects focused on improving diagnostics and developing treatments for rare muscle diseases, especially the muscular dystrophies caused by mutations in the components of the Dystrophin Glycoprotein Complex (DGC).
I led a project examining the functional consequences of all possible small-size variants of alpha-dystroglycanopathy related genes in a high-throughput manner. In this project, I developed a simple, cost-effective, and adaptable workflow for high-throughput functional screening of variants, which was named Saturation mutagenesis-reinforced Functional assays (SMuRF). We have applied SMuRF and screened all possible coding SNVs of FKRP and LARGE1. The Lek lab is expanding SMuRF by incorporating different saturation mutagenesis methods and different functional assays and will apply it to more genes.
I conducted a project to develop a personalized gene therapy which employed the CRISPR-activation technology to up-regulate the cortical dystrophin in patients who lack the muscle isoform. I designed and cloned the CRISPRa construct used to make the clinical drug and performed the pre-clinical studies to test it both in cell lines and mouse models. The Investigational New Drug (IND) Application for the clinical trial of this project obtained “Safe to Proceed” from FDA (IND, 28497; https://beta.clinicaltrials.gov/study/NCT05514249).
I also worked on developing novel systemic delivery systems, including engineered AAV serotypes and antibody-based delivery, to increase safety and delivering efficiency to the muscular tissues.
2. The Internship in UCSD and My Undergraduate Thesis Honor Study (2017-2018)
In 2017, I did a summer internship in Dr. Bing Ren’s Lab at University of California, San Diego, which was part of the Ludwig Institute for Cancer Research. I worked with Dr. Yarui Diao and learned high-throughput CRIPSR gene screening technologies. I did a GeCKO (Genome-Scale CRISPR Knock-Out) screen to identify genes related to hESC pluripotency. I also finished my thesis honor study in the Ren lab. The aims of my thesis were developing the Whole-Genome CREST-seq (cis-regulatory element scan by tiling-deletion and sequencing) technology and identifying drug targets and tumorigenesis genes. During this project, I received a 30,000 CNY USTCIF Overseas Exchange funding.
3. The Plant Molecular Biology Lab at USTC (2016-2017)
I worked in the PMB Lab for 1.5 yrs. Under Prof. Chengbin Xiang’s supervising, I finished my Student Researching Program with a score A. The title of my project was Expression Pattern Analysis of NLPs in Arabidopsis thaliana. In that project, I concluded the expression pattern of 7 members of 9 AtNLP proteins.
4. The International Genetically Engineering Machinery Competition (iGEM) (2015-2016)
In 2015, our project aimed at detecting trace antibiotics in water bodies by using engineered bacteria. In 2016, I was the student leader of Team USTC; we constructed a protein-level switch based on yeast prion.
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