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The Lefler Center

for the study of Neurodegenerative Diseases

Bence Gyorgy, MD, PhD

Research Fellow, The Corey Lab, Department of Neurobiology, Harvard Medical School

2017 Lefler Fellow

1.  What motivated you to come to Harvard Medical School?

I wanted to learn more about gene therapy and gene delivery vectors (such as viruses and cell-derived lipid vesicles, i.e. exosomes), so I decided to apply for a postdoctoral position in the labs of Dr. Xandra Breakefield and Dr. Casey Maguire at the Massachusetts General Hospital (MGH). I was quite surprised to learn that I was offered a position at Harvard Medical School, Department of Neurobiology with Dr. David Corey to work on gene therapy for hearing in collaboration with Dr. Breakefield and Dr. Maguire. Since then, I’m working both at MGH and HMS in a highly collaborative team pursuing the development of novel genetic based therapies for hereditary hearing loss and other neurodegenerative diseases.

2. What is your current area of research?

I’m working on developing therapeutic strategies against recessive and dominant forms of genetic hearing loss. Previously we have shown that a novel type of complex gene delivery vector (adeno-associted virus (AAV) vectors in complex with exosomes) was able to deliver a therapeutic gene to sensory hair cells of the inner ear – thereby restoring hearing and improving balance function in a mouse model of human deafness. Currently we are working on continuing this gene therapy approach in order to translate the approach to the clinic by refining our AAV strategy and testing gene delivery in larger animals. Also, we are applying in vivo genome editing platforms to overcome the dominant negative effects of a mutant protein to stop degeneration of sensory hair cells in the cochlea. Furthermore, I’m interested in creating novel tools to define in vivo genome-wide specificity and precision of CRISPR/Cas9 and other genome editing nucleases.

3. What are your research goals?

My research goals in general are to develop genetic therapies that could be ultimately applied in clinical practice. I’m also very interested to explore novel platforms for genetic manipulation (e.g. viruses, non-viral carriers, proteins that manipulate DNA, RNA and epigenome) and I think the incredibly diverse world of microbes is almost an endless source for discovery.

4. What are your career goals?

I would like to become a clinician scientist in the future and focus on translational research. I think being a Lefler fellow would enable me to get a deeper understanding in basic neuroscience. I believe that for the future generation of doctors – particularly who work in the field of translational research –  the deep understanding in basic sciences cannot be overemphasized. I am very fortunate to be able to spend several years in research that would allow me to build on these experiences in the future. I’m hoping to be able to stay in an academial medical center later and to do research, help patients and also teach the younger generation.

5. What do you think is the next frontier in brain research?

I think the next frontier will be to understand how genes orchestrate the development of the brain and how they determine connectivity, plasticity, behavior and disease. Also, it will be interesting to get an insight how genetic manipulation in the developed brain affect plasticity and behavior in adults. A huge step forward (I think within 10 years!) will be to discover other components of DNA editing systems in order to create precise genetic changes in in vivo, for example in neurons. I think we will see major breakthroughs in the 21st century with respect to the treatment of genetic disease and I strongly believe that gene therapy in humans to alleviate suffering and combat currently incurable diseases will soon become a reality.

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