Content
Special Interview with Dr. Henry Lee
Recently, the Editorial Office of Journal of Translational Genetics and Genomics interviewed Dr. Henry Lee, research associate in Neurology at Boston Children’s Hospital, Boston, Massachusetts, USA.
In the interview, Dr. Lee discussed his transition from fundamental neuroscience research toward translational medicine. He explained how his interest in SSADHD emerged from the close connection between his basic research on GABAergic inhibition and the pathophysiology of the disease. The recognition that a single genetic defect can disrupt a well-defined biochemical pathway also provided an opportunity to explore gene therapy as a potential treatment for a disorder for which no gene therapy is currently available.
Dr. Lee further discussed the major challenges associated with developing gene therapies for neurological rare diseases. Studies in mouse models demonstrated that SSADHD-associated neurological abnormalities can be substantially reversed when the relevant gene is restored throughout the brain. These findings highlighted a central challenge for therapeutic development: achieving sufficiently broad and efficient gene delivery across the central nervous system.
Watch the full interview with Dr. Henry Lee:
Interview Questions:
Q1. Dr.Lee, you have long been dedicated to studying neuronal inhibition, brain development, and neurological disorders, and have increasingly focused on translational therapies for the rare disease SSADHD. What inspired you to turn your attention to this disorder, and how do you see fundamental neuroscience research contributing to the development of treatments for rare diseases?
Q2. For neurological rare diseases such as SSADHD, what are the key challenges in developing gene therapies that can effectively reach the central nervous system, and what breakthroughs are you most looking forward to in this field?
About the Interviewee:

Henry Lee is a Research Associate in Neurology at Boston Children’s Hospital. His research has focused on neuronal inhibition, brain development, and the molecular mechanisms underlying neurological disorders, with particular interests in GABAergic neurotransmission and its role in brain function and disease.
Dr. Lee received his Doctor of Philosophy in Neuroscience, Physiology and Pharmacology and previously completed a Master of Philosophy in Molecular Neurobiology. He also holds a First Class Honors Bachelor of Science degree in Biochemistry.
His research has increasingly incorporated translational approaches to rare neurological diseases, including SSADHD. By combining fundamental neuroscience with molecular and genetic approaches, his work aims to elucidate disease mechanisms and develop potential therapeutic strategies for patients with rare disorders.
Representative Research Achievements
- 2022 Young Investigator Award
- 2021 R21 Award
- 2020 Research Program Grant
- 2010 Croucher Foundation Post-doctoral Fellowship
- 2009 Founder’s Affiliate AHA Post-doctoral Fellowship
- 2007 AES Pre-doctoral Research Training Fellowship
- 2000 Sir Edward Youde Memorial Scholarship
- 1999 The George K Lee Foundation Scholarship
- 1998 Wu Ti Hsien Science and Education Foundation Fund Scholarship
He has also received academic distinctions in chemistry and biology and has a strong background in biochemistry, molecular neurobiology, neuroscience, physiology, and pharmacology.
Editor: Iris Yang
Language Editor: Catherine Yang
Production Editor: Xingyue Luo
Respectfully Submitted by the Editorial Office of Journal of Translational Genetics and Genomics





