Hiroyuki Nakai, MD, PhD

Bridge Grant Pilot Program

Institution: Oregon Health & Science University

How would you sum up your overall research focus in one sentence?

My overall research focuses on elucidating the biology of adeno-associated virus (AAV) vectors and applying this knowledge to develop innovative gene therapy strategies.

Provide a brief overview of the research project you will conduct with help from the grant.

Our laboratory pioneered AAV vector-mediated gene delivery to the kidney and developed effective strategies for delivering therapeutic cargos to podocytes and renal tubules in vivo. This grant will support preclinical validation of our approach to treat Alport syndrome using novel AAV capsid-based vectors showing substantially enhanced podocyte transduction and able to express full-length COL4A5 protein in Alport mice.

What inspired you to focus your work in this area?

Gene delivery to therapeutically relevant kidney cells, such as renal tubules and podocytes, has historically been considered highly challenging. However, our laboratory discovered in the late 2010s that this dogma does not hold true, inspiring us to establish a new research initiative focused on developing novel AAV vector-mediated gene therapy for kidney diseases.

What impact do you hope your research will have on people with kidney diseases?

Many kidney diseases currently lack effective disease-modifying therapies, including genetic kidney diseases that progress to renal failure early in life. My goal is to leverage the knowledge about the biology of AAV vectors and gene therapy technologies our laboratory has developed to transform therapeutic approaches for kidney diseases.

What has surprised you most about your career?

What has surprised me most is that my scientific curiosity remains boundless and my passion for research continues to grow stronger over time. I feel fortunate that I can sustain a fulfilling career while pursuing these endless questions that fascinate me.

What are the major challenges facing nephrology research today?

Limited funding resources continues to the major challenge, particularly for junior investigators.

In one sentence, please describe the importance of having grant funding available through KidneyCure.

Due to substantial delays in NIH funding, our research program was at risk of disruption, but this grant rescued us from that crisis.

Something you may not know about me is…

I may carry a short sleeper gene, having required only 3 to 4 hours of sleep per night for more than 45 years.

In my free time I like to…

Sip coffee at Starbucks while writing codes for AI tools for fun, and play piano.