Rajesh C. Rao, M.D.

Rajesh Rao, M.D.

Leonard G Miller Professor of Ophthalmology and Visual Sciences
Associate Professor of Ophthalmology and Visual Sciences
Associate Professor of Pathology
Associate Professor of Human Genetics
Director of Retina, Section of Ophthalmology, Surgery Service, VA Ann Arbor Healthsystem

Brehm 8333
1000 Wall St
Ann Arbor MI 48109-5714



The goal of my research is to contribute foundational insights that mediate how epitranscriptomic mechanisms control retinogenesis, using pluripotent stem cell models. I have over 20 years of experience in stem cell biology. In 2013, I was the youngest awardee and only trainee to be selected among the 10 winners (out of ~500) in the National Eye Institute Audacious Goals Challenge. After joining U. Michigan as an K12 Scholar, I have earned nationally competitive grants, including NIH K08 and Research to Prevent Blindness career development awards. I have published 99 papers in stem cell biology, epigenetics, and ophthalmology. I have made seminal findings that link, for the first time, histone methylation to human vision diseases by discovery of novel biomarkers and actionable targets (Lab Invest 2015, JAMA Oncology 2016, Mod Path 2016, Cell Oncol 2018, Cell Reports 2020, Stem Cell Reports 2021). In December 2018, I became one of the youngest faculty to be named to a full endowed professorship at the U. of Michigan (Leonard G. Miller Professorship). In September 2021, I was promoted to Associate Professor with Tenure.

Honors & Awards

  • 2022                       Career Advancement Award, Research to Prevent Blindness
  • 2018                      Appointed Leonard G. Miller Professor of Ophthalmology & Visual Sciences (Endowed Chair)
  • 2017                      Young Physician Scientist Award, American Society of Clinical Investigation
  • 2017-2020          Elected to Senate Assembly, University of Michigan, Ann Arbor
  • 2016-2019          E. Matilda Ziegler Foundation for the Blind Inc. Early Career Faculty Grant
  • 2016-2017          Alcon Research Institute Young Investigator Grant
  • 2015-2020          Appointed Leslie and Abigail Wexner Emerging Scholar, Taubman Medical Research Institute
  • 2015-2017          Knights Templar Eye Foundation Career Starter Grant (Renewal Awarded)
  • 2015-2016          Biointerfaces Institute-Kellogg Eye Center Challenge Grant, University of Michigan, Ann Arbor
  • 2014-2018          Career Development Award, Research to Prevent Blindness
  • 2013-2016          Inaugural NEI/NIH K12 Scholar, University of Michigan
  • 2013                      Winner, NEI Challenge to Identify Audacious Goals in Vision Research & Blindness Rehabilitation
  • 2012-2013          Heed Fellow, Heed Ophthalmic Foundation
  • 2010                      Association for Research in Vision and Ophthalmology (ARVO) Foundation for Eye Research /
  • Retina Research Foundation / Joseph M. and Eula C. Lawrence Travel Scholarship
  • 2007                      Honors Thesis, Yale University School of Medicine
  • 2007                      Department of Laboratory Medicine Thesis Award, Yale University School of Medicine
  • 2007                      Norma Bailey Berniker Prize, Yale University School of Medicine
  • 2007                      Farr Scholar, Yale University School of Medicine
  • 2007                      Alpha Omega Alpha Honor Medical Society, Yale University School of Medicine
  • 2002                      Finalist, Paul & Daisy Soros Fellowship for New Americans
  • 2001                      Special Achievement Award, National Institutes of Health



  • University of Wisconsin-Madison, BS, 08/2000, Molecular Biology
  • Howard Hughes Medical Institute – NIH Research Scholars Program, Bethesda/Chevy Chase, MD Research Scholar, 06/2006, Stem Cell Biology
  • Yale University School of Medicine, New Haven, CT, MD, 05/2007, Medicine
  • McGaw Medical Center of Northwestern University, Evanston, IL, Intern, 06/2008, Transitional Year
  • Massachusetts Eye and Ear Infirmary, Harvard Medical School, Boston, MA, Resident, 06/2011, Ophthalmology
  • Schepens Eye Research Institute, Harvard Medical School, Boston, MA (concurrent with residency), Research Fellow, 06/2011, Epigenetics of Retinal Development
  • Barnes Retina Institute, Washington University School of Medicine, St. Louis, MO, Clinical Heed Fellow, 06/2013, Vitreoretinal Diseases and Surgery

Published Articles or Reviews

  1. Rao RC, Boyd J, Padmanabhan R, Chenoweth JG, McKay RD. Efficient serum-free derivation of oligodendrocyte precursors from neural stem cell-enriched cultures. Stem Cells 27(1): 116-25, 2009.
  2. Rao RC, Tchedre KT, Malik MT, Coleman N, Fang Y, Marquez VE, Chen DF. Invest Ophthalmol Vis Sci . 2010 Dec;51(12):6784-92. PMCID: PMC3055777
  3. Jakobiec FA, Zakka FR, D’Amato RJ, DeAngelis MM, Walton DS, Rao RC. Unilateral sporadic retinal dysplasia: results of histopathologic, immunohistochemical, chromosomal, genetic and VEGF-A analyses. Journal of the American Association for Pediatric Ophthalmology and Strabismus 15(6):579-86, 2011. PMCID: PMC4242509
  4. Choudhry N, Rao RC. Valsalva retinopathy. The New England Journal of Medicine. 20;370(8):e13,2014.
  5. Khan M, Walters LL, Li Q, Thomas DG, Miller JML, Zhang Q, Sciallis AP, Liu Y, Dlouhy BJ, Fort PE, Archer SM, Demirci H, Dou Y, Rao RC. Characterization and pharmacologic targeting of EZH2, a fetal retinal protein and epigenetic regulator, in human retinoblastoma. Laboratory Investigation. 95(11):1278-90, 2015. PMCID: PMC4626270
  6. Rao RC, Gragoudas ES. Choroidal metastases from EML4-ALK positive non-small cell lung adenocarcinoma. Journal of Clinical Oncology. Oct 20;33(30):e112-4, 2015.
  7. Rao RC, Dou Y. Hijacked in cancer: the KMT2 (MLL) family of methyltransferases. Nat Rev Cancer. 2015 Jun;15(6):334-46
  8. Rao RC, Chan MP, Andrews CA, Kahana A. EZH2, proliferation rate, and aggressive tumor subtypes in cutaneous basal cell carcinoma. JAMA Oncology. 2016; 2(7):962-3.
  9. Zhang H, Gayen S, Xiong J, Zhou B, Shanmugam AK, Sun Y, Karatas H, Liu L, Rao RC, Wang S, Nesvizhskii AI, Kalantry S, Dou Y. MLL1 inhibition reprograms epiblast stem cells to naive pluripotency. Cell Stem Cell. 2016; 18(4):481-94.
  10. Choudhry N, Golding J, Manry MW, Rao RC. Ultra-widefield steering technique-based SD-OCT imaging of the far retinal periphery. Ophthalmology. 123(6):1368-74, 2016.
  11. Cani AK, Soliman M, Hovelson DH, Liu CJ, McDaniel AS, Haller MJ, Bratley JV, Rahrig SE, Li Q, Briceño CA, Tomlins SA, Rao RC. Comprehensive genomic profiling of orbital and ocular adnexal lymphomas identifies frequent alterations in MYD88 and chromatin modifiers: new routes to targeted therapies. Modern Pathology. 29(7):685-97, 2016. PMCID: PMC4925176.
  12. Yan N, Cheng L, Cho K, Malik MT, Xiao L, Guo C, Yu H, Zhu R, Rao RC, Chen DF. Postnatal onset of retinal degeneration by loss of embryonic Ezh2 repression of Six1. Scientific Reports. 2016 Sep 28;6:33887.
  13. Zhou B, Wang J, Lee S, Xiong J, Ma P, Sun Y, Rao RC, Hess JL, Dou Y. PRDM16 suppresses MLL1r leukemia via intrinsic histone methyltransferase activity. Molecular Cell. 62(2):222-236, 2016. PMCID: PMC5061593.
  14. Cani AK, Hovelson DH, Demirci H, Johnson MW, Tomlins SA, Rao RC. Next generation sequencing of vitreoretinal lymphomas from small-volume intraocular liquid biopsies: new routes to targeted therapies. Oncotarget. 2017; 8(5):7989-7998.
  15. Li Q, Mao, F, Zhou B, Huang Y, Zou Z, denDekker A, Xu J, Hou S, Liu J, Dou Y, Rao RC. p53 integrates temporal WDR5 inputs during neuroectoderm and mesoderm differentiation of mouse embryonic stem cells. Cell Reports. 2020 Jan 14;30(2):465-480. PMCID: PMC7024586.
  16. Rao RC, Dlouhy BJ, Capell BC, Akeju O. The endangered physician-scientist and COVID-19. Cell Rep Med. 2021 Feb 16;2(2):100190. PMID: 33521693  PMCID: PMC7832285
  17. Basinski BW, Balikov DA, Aksu M, Li Q, Rao RC. Ubiquitous Chromatin Modifiers in Congenital Retinal Diseases: Implications for Disease Modeling and Regenerative Medicine. Trends Mol Med . 2021 Apr;27(4):365-378
  18. Balikov DA, Bixby DL, Rao RC. Retinal haemorrhage as a complication of blastic plasmacytoid dendritic cell neoplasm. Lancet Haemotology. Sep;8(9):e670, 2021
  19. Zhao PY, Ji S, Newman-Casey PA, Andrews CA, Lonardi A, Bennett OM, Dinh DQ, Talwar N, Hutton DW, Temple S, Stern JH, Rao RC. Assessing patient perception of risk in ocular stem cell therapies. Stem Cell Reports. Oct 12;16(10):2415-2421, 2021. PMCID: PMC8514963
  20. Rao RC, Stern JH, Temple S. The eyeball's connected to the brain ball. Cell Stem Cell . 2021 Oct 7;28(10):1675-1677. 
  21. Li Q, Huang Y, Xu J, Mao, F, Zhou B, Sun L, Basinski BW, Aksu M, Liu Jie, Dou Y, Rao RC. p53 inactivation unmasks histone methylation-independent WDR5 functions that drive self-renewal and differentiation of pluripotent stem cells. Stem Cell Reports. Nov 9;16(11):2642-2658, 2021. PMCID: PMC8581203.

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