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Kiran Musunuru, MD, PhD, MPH, ML, MRA

Kiran Musunuru

Kiran Musunuru, MD, PhD, MPH, ML, MRA

(He/Him)

Kiran Musunuru, MD, PhD, MPH, ML, MRA, is Co-Director of the Penn Medicine/CHOP Orphan Disease Center.

Locations: Main Building

About Kiran Musunuru, MD, PhD, MPH, ML, MRA

An actively practicing cardiologist and committed teacher, Kiran Musunuru, M.D., Ph.D., M.P.H., M.L., M.R.A., is the Barry J. Gertz Professor for Translational Medicine in the Perelman School of Medicine at the University of Pennsylvania. His research focuses on the development of novel gene editing therapies for the treatment of cardiovascular and metabolic diseases. He is a recipient of the Presidential Early Career Award for Scientists and Engineers from the White House, the Pennsylvania Governor's Keystone Award, the American Heart Association's Award of Meritorious Achievement and Joseph A. Vita Award, the American Philosophical Society's Judson Daland Prize for Outstanding Achievement in Clinical Investigation, the American Federation for Medical Research's Outstanding Investigator Award, Harvard University's Fannie Cox Prize for Excellence in Science Teaching, and the University of Pennsylvania's Jane M. Glick Graduate Student Teaching Award. He recently served as Editor-in-Chief of the scientific journal Circulation: Genomic and Precision Medicine and is the current Editor-in-Chief of The American Journal of Human Genetics. He is author of The CRISPR Generation: The Story of the World's First Gene-Edited Babies and Genome Editing: A Practical Guide to Research and Clinical Applications.

Titles

Barry J. Gertz Professor for Translational Research

Co-Director, Penn Medicine/CHOP Orphan Disease Center

Director, Genetic and Epigenetic Origins of Disease Program, Cardiovascular Institute

Scientific Director, Center for Inherited Cardiovascular Disease, Perelman School of Medicine at the University of Pennsylvania

Awards and Honors

Presidential Early Career Award for Scientists and Engineers from the White House

Pennsylvania Governor's Keystone Award

American Heart Association's Award of Meritorious Achievement

Joseph A. Vita Award

American Philosophical Society's Judson Daland Prize for Outstanding Achievement in Clinical Investigation

American Federation for Medical Research's Outstanding Investigator Award

Harvard University's Fannie Cox Prize for Excellence in Science Teaching

University of Pennsylvania's Jane M. Glick Graduate Student Teaching Award

Education & training

Graduate Degree

PhD - The Rockefeller University, New York, NY

MPH - Johns Hopkins Bloomberg School of Public Health, Baltimore, MD

ML - University of Pennsylvania Law School, Philadelphia, PA

MRA - Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA

Medical Degree

MD - Weill-Cornell Medical College, New York, NY

Publications

Publications

2025

Gurevitz C, Bajaj A, Khera AV, Do R, Schunkert H, Musunuru K, Rosenson RS.: Gene therapy and genome editing for lipoprotein disorders. Eur Heart J Jul 2025.

Shuvaev VV, Tam YK, Lee BW, Myerson JW, Herbst A, Kiseleva RY, Glassman PM, Parhiz H, Alameh MG, Pardi N, Muramatsu H, Shuvaeva TI, Arguiri E, Marcos-Contreras OA, Hood ED, Brysgel TV, Nong J, Papp TE, Eaton DM, Riley R, Palanki R, Musunuru K, Brenner JS, Mitchell MJ, Ferrari VA, Mui BL, Semple SC, Weppler SA, Atluri P, Margulies KB, Weissman D, Muzykantov VR.: Systemic delivery of biotherapeutic RNA to the myocardium transiently modulates cardiac contractility in vivo. Proc Natl Acad Sci U S A 122: e2409266122, Jul 2025.

Musunuru K, Grandinette SA, Wang X, Hudson TR, Briseno K, Berry AM, Hacker JL, Hsu A, Silverstein RA, Hille LT, Ogul AN, Robinson-Garvin NA, Small JC, McCague S, Burke SM, Wright CM, Bick S, Indurthi V, Sharma S, Jepperson M, Vakulskas CA, Collingwood M, Keogh K, Jacobi A, Sturgeon M, Brommel C, Schmaljohn E, Kurgan G, Osborne T, Zhang H, Kinney K, Rettig G, Barbosa CJ, Semple SC, Tam YK, Lutz C, George LA, Kleinstiver BP, Liu DR, Ng K, Kassim SH, Giannikopoulos P, Alameh MG, Urnov FD, Ahrens-Nicklas RC.: Patient-specific in vivo gene editing to treat a rare genetic disease. N Engl J Med 392: 2235-2243, Jun 2025.

Urnov F, Kassim S, Musunuru K, Liu D, Lee A, Barrera L, Stetkiewicz P, Bruno J, Hewitt M, Lister T, Malech H, Gasch L, Diver M, Gertler N, Grignon F, Le A, Lehmicke M, Almendro-Navarro V, Lembong J.: Advancing gene-editing platforms to improve the viability of rare-disease therapeutics: key insights from a 2024 Scientific Exchange hosted by ARM, ISCT, and Danaher. Cytotherapy Jun 2025.

Musunuru K, Urnov F.: Moving Therapeutic Genome Editing into Global Clinical Trials and Medicine. CRISPR J May 2025.

Musunuru K.: A milestone for gene-editing therapies. N Engl J Med 392: 508-509, Jan 2025.

Allyse MA, Agam P, Bombard Y, Feys R, Horstmann M, Kokayi A, Isasi R, Meagher KM, Michie M, Musunuru K, Ormond KE, Riggan KA, Yap JQ.: Building better medicine: translational justice and the quest for equity in US healthcare. Am J Bioeth 2025.

Kassim SH, Urnov F, Musunuru K, Lee A, Barrera L, Stetkiewicz P, Bruno J, Hewitt M, Lister T, Malech H, Gasch L, Diver M, Gertler N, Grignon F, Le A, Lehmicke M, Horwitz EM, Liu DR, Lembong J, Almendro-Navarro V.: Platform solutions for commercial challenges to expanding patient access and making gene editing sustainable. Nat Biotechnol 2025.

2024

IGVF Consortium: Deciphering the impact of genomic variation on function. Nature 633: 47-57, Sep 2024.

Riggan KA, Feys R, Kokayi A, Meagher KM, Michie M, Musunuru K, Ormond KE, Schelhaas AJ, Yap JQ, Isasi R, Allyse MA.: Grounded in reality: integrating community values and priorities of end users in human gene editing. Am J Bioeth 24: 43-45, Aug 2024.

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