Gabsang Lee, PhD

Dr. Lee is a Professor at Johns Hopkins University School of Medicine, where he is studying stem cells created from the cells of patients with rare diseases like Familial Dysautonomia, Charcot-Marie-Tooth disease, and muscular dystrophy in order to investigate their pathology and develop new treatments and therapies. During his time as a NYSCF – Druckenmiller Fellow at MSKCC, he established a novel methodology for direct derivation and prospective isolation of neural crest cells derived from human pluripotent stem cells.

Dr. Lee aimed to use stem cells to model specific genetic diseases such as familial dysautonomia (FD), a rare and fatal disorder primarily affecting nerve cells. By creating stem cell models, he was able to pursue a systematic drug discovery effort to find potential therapeutic options. Using a sophisticated analytical approach, he screened 6,912 chemical compounds, composed of FDA-approved drugs and natural products, and identified 8 validated hits, which demonstrated the feasibility of performing drug screening on stem cell models. He is continuing to work on rare diseases in his independent position. He completed his postdoctoral studies at MSKCC in Dr. Lorenz Studer’s lab.

• PubMed Profile
• Dr. Lee's Lab Website

• Neurobiology

• NYSCF Innovator Shows Zika's Effects on Peripheral Nervous System
• NYSCF Innovators Use Stem Cells to Investigate Familial Dysautonomia
• Using Stem Cells to Study the Nervous System
• NYSCF - Robertson Investigators Study and Quantify Pluripotency
• NYSCF - Robertson Investigator Reprograms Skin Cells to Mimic Rare Disease