Ronak Lakhia, MD
Ronak Lakhia, MD
2021 Carl W. Gottschalk Research Scholar Grant
Institution: University of Texas Southwestern Medical Center
Project Title: Role of Cis-regulatory Elements in Autosomal Dominant Polycystic Kidney Disease
How would you sum up your research in one sentence?
Our laboratory seeks to understand and manipulate the metabolic and epigenetic aberrancies that underlie ADPKD (Autosomal Dominant Polycystic Kidney Disease) to find precise mechanisms to slow cyst growth.
Provide a brief overview of the research you will conduct with help from the grant.
Transcriptional dysregulation is a prominent feature of polycystic kidneys. We have discovered that the epigenetic landscape of ADPKD kidneys is markedly altered compared to normal kidneys. In addition, we have identified and characterized several distinct regulatory elements that promote aberrant gene expression in ADPKD models. This award will allow our laboratory to use a series of genetic approaches to delete or re-direct enhancers away from their target promoter as means to reduce pathogenic gene expression. These studies will provide proof of principle of the feasibility and efficacy of targeting the epigenome to slow kidney cyst growth.
What inspired you to focus your research in this area?
Since I was an undergraduate, I have been fascinated by genetic disorders and gene regulation. In addition, as a nephrology fellow I was impressed by the spectrum of disease burden we see in individuals with ADPKD even within families. I am privileged to be able to couple my basic science and clinical interests this early in my career.
What impact do you hope your research will have on patients?
ADPKD is among the most common monogenetic disorders in the United States and treatment options are limited. We hope that the discoveries from this project will identify novel targets and approaches for drug development for the treatment of ADPKD.
What are your career goals at the end of the grant period? Five years out? Ten years out?
My long-term goals are to become an expert in the pathophysiology of ADPKD and develop novel approaches to correct the fundamental aberrancies that promote cyst growth.
What has surprised you most about your career?
It is surprising to me how generous senior investigators are with their time to help you develop as a scientist. I have been fortunate to train and start my career at an institution that is genuinely devoted to my professional development.
What advice would you give to others to encourage them to apply for this grant funding?
The KidneyCure TIG grant is an excellent opportunity for early/junior investigators. Fellows and post-docs in the latter half of their training should be aware of this opportunity, actively plan an application, and discuss with their mentor timing of submission.
In my free time…
I like to spend time with my family cooking and gardening.