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Molecular Medicine


Laura Niedernhofer, M.D., Ph.D.

Associate Professor
Department of Molecular Medicine
Florida Campus
Laboratory Website
(561) 228-2142

Scripps Research Joint Appointments

Faculty, Graduate Program

Research Focus

My research career has been dedicated to investigating the impact of DNA damage on the structure of DNA, cell function and organism health. The DNA in each of our cells is damaged thousands of times per day by exposure to environmental factors, dietary components, chemotherapeutic agents and even endogenous by-products of normal metabolism. Studying patients with rare diseases caused by inherited defects in DNA repair provides important insight into the consequences of DNA damage. These patients have a dramatically increased risk of cancer and age prematurely. We engineered mouse models of these genome instability syndromes as a sensitive tool to test hypotheses about how DNA damage promotes cancer and aging.

Our current research program at Scripps Florida focuses on (1) discovering the mechanism by which endogenous and environmental genotoxic stress promotes aging and age-related degenerative diseases; (2) identifying the mechanism by which cancer chemotherapeutic agents, including cisplatin, stop tumor growth with the goal of improving outcomes in patients with solid tumors; and (3) determining if changes in the capacity to repair DNA contributes to cancer risk, cancer recurrence after therapy, and/or human lifespan/healthspan.


B.S., Chemistry, Duke University, Durham, NC, 1985
M.S., Physiology, Georgetown University School of Medicine, Washington, D.C., 1990
Ph.D., Biochemistry, Vanderbilt University School of Medicine, Nashville, TN, 1996
M.D., Vanderbilt University School of Medicine, Nashville, TN, 1998

Professional Experience

1982 – 1984: Department of Neurooncology, Research Assistant, Duke University, Durham, NC
1985 – 1989: Department of Toxicology, Research Assistant, Massachusetts Institute of Technology, Cambridge, MA
2003 – 2009: Department of Microbiology and Molecular Genetics, Assistant Professor, University of Pittsburgh School of Medicine; Molecular and Cellular Biology Program, University of Pittsburgh Cancer Institute
2009 – 2012: Department of Microbiology and Molecular Genetics, Associate Professor, University of Pittsburgh School of Medicine; Molecular and Cellular Biology Program, University of Pittsburgh Cancer Institute
2012 – Present: Department of Metabolism and Aging, Associate Professor, The Scripps Research Institute, Jupiter, FL

Awards & Professional Activities

June 1992 – May 1994 Medical Scientist Training Program Traineeship, NIH
June 1994 – May 1996 Predoctoral Traineeship on the Breast Cancer Training Grant, DOD
1998 Elected to Sigma Xi
June 1999 – Jan. 2000 National Science Foundation International Research Fellow Award
Jan. 2000 – Dec. 2002 American Cancer Society Post-doctoral fellowship
2005 PNC Foundation Innovation Award
2005 – 2006 Hillman Foundation Fellow
2005 – 2009 Ellison Medical Foundation New Scholar in Aging
2007 – 2012 Outstanding New Environmental Scientist, NIEHS
2008 Elected to the American Society for Clinical Investigation
2009 Hillman Fellow for Innovative Cancer Research
2010 Chancellor's Distinguished Research Award
2013 Scripps Florida Society of Research Fellows Mentorship Award
2013 Hart Family Award for Science and Science Advocacy

Editorial Boards
2003 – Present DNA Repair
2009 – 2012 Pitt Med Magazine
2010 – 2015 Associate Editor: DNA Repair
2012 – present Guest Editor PLoS One
2016 – present Editorial Board Experimental Gerontology
2016 – present Board of Consulting Editors of Journal of Clinical Investigation Insights

Professional Activities
2004-2009 Eukaryotic Molecular Genetics (MSBMG 2520)
Biochemistry and Molecular Genetics graduate course
University of Pittsburgh School of Medicine;

2004-2009 Chromosomes and Human Disease (HUGEN 2031)
Department of Human Genetics
University of Pittsburgh, School of Public Health

2005-2010 M.S.T.P. Research Basis of Medical Knowledge
University of Pittsburgh School of Medicine

2006-2010 Current Topics in Molecular, Cell and Developmental Biology (BIOSCI 2011)
Department of Biological Sciences, University of Pittsburgh

2006-2012 DNA Repair: from biochemistry to human disease (MSBMG 3530; MSMPHL 3330)
Biochemistry and Molecular Genetics graduate course
University of Pittsburgh School of Medicine;
Course co-director and lecturer (5 lectures)

2008-2012 DNA Repair Seminar (MSBMG 3535; MSMPHL 3335; MSCBMP 3835)

2010-2012 Biology and Physiology of Aging (EPIDEM 2980)

2010-2011 Physicians Scientist Training Program summer research course

2012 Model Organisms (MSMGDB 2535)

2013-2014 Seminar Series for Summer Undergraduate Interns 
Scripps Florida  

2013-2016 Investigations in Molecular Biology
TSRI Graduate Program

2013-2016 Frontiers in Aging and Regeneration Research

Xavier University and Morehouse College
2015 Cancer Biology

Community Activities
2004 – present Volunteer Camp Sundown for children with xeroderma pigmentosum
2014 – present Women's Foundation of Palm Beach County
2014 – present Palm Beach County Commission on the Status of Women
2015 – present Trustee of the North Palm Beach County Chamber of Commerce for Scripps
2015 – present Scripps Faculty Philanthropy Committee
2015 – present Scripps Community Relations Committee

Selected References

Burd CE, Gill MS, Niedernhofer LJ, Robbins PD, Austad SN, Barzilai N, Kirkland JL. Barriers to the Preclinical Development of Therapeutics that Target Aging Mechanisms. (2016) J Gerontol A Biol Sci Med Sci. PubMed PMID: 27535964; PubMed Central PMCID: PMC5055650.

Niedernhofer LJ, Kirkland JL, Ladiges W. Molecular pathology endpoints useful for aging studies. (2016) Ageing Res Rev. Review. PubMed PMID: 27721062.

Zhu Y, Tchkonia T, Fuhrmann-Stroissnigg H, Dai HM, Ling YY, Stout MB, Pirtskhalava T, Giorgadze N, Johnson KO, Giles CB, Wren JD, Niedernhofer LJ, Robbins PD, Kirkland JL. Identification of a novel senolytic agent, navitoclax, targeting the Bcl-2 family of anti-apoptotic factors. (2016) Aging Cell. 15(3):428-35. PubMed PMID: 26711051; PubMed Central PMCID: PMC4854923.

Gurkar AU, Niedernhofer LJ. Comparison of mice with accelerated aging caused by distinct mechanisms. (2015) Exp Gerontol. 68:43-50. Review. PubMed PMID: 25617508; PubMed Central PMCID: PMC4464936.

Zhu Y, Tchkonia T, Pirtskhalava T, Gower AC, Ding H, Giorgadze N, Palmer AK, Ikeno Y, Hubbard GB, Lenburg M, O'Hara SP, LaRusso NF, Miller JD, Roos CM, Verzosa GC, LeBrasseur NK, Wren JD, Farr JN, Khosla S, Stout MB, McGowan SJ, Fuhrmann-Stroissnigg H, Gurkar AU, Zhao J, Colangelo D, Dorronsoro A, Ling YY, Barghouthy AS, Navarro DC, Sano T, Robbins PD, Niedernhofer LJ, Kirkland JL. The Achilles' heel of senescent cells: from transcriptome to senolytic drugs. (2015) Aging Cell. 14(4):644-58. PubMed PMID: 25754370; PubMed Central PMCID: PMC4531078.

Vaezi AE, Bepler G, Bhagwat NR, Malysa A, Rubatt JM, Chen W, Hood BL, Conrads TP, Wang L, Kemp CE, Niedernhofer LJ. Choline phosphate cytidylyltransferase-α is a novel antigen detected by the anti-ERCC1 antibody 8F1 with biomarker value in patients with lung and head and neck squamous cell carcinomas. (2014) Cancer. 120(12):1898-907. PubMed PMID: 24692084; PubMed Central PMCID: PMC4047200.

Chen Q, Liu K, Robinson AR, Clauson CL, Blair HC, Robbins PD, Niedernhofer LJ, Ouyang H. DNA damage drives accelerated bone aging via an NF-κB-dependent mechanism. (2013) J Bone Miner Res. 28(5):1214-28. PubMed PMID: 23281008; PubMed Central PMCID: PMC3662975

Cho JS, Kook SH, Robinson AR, Niedernhofer LJ, Lee BC. Cell autonomous and nonautonomous mechanisms drive hematopoietic stem/progenitor cell loss in the absence of DNA repair. (2013) Stem Cells. 31(3):511-25. PubMed PMID: 23097336; PubMed Central PMCID: PMC3582850.

Clauson C, Schärer OD, Niedernhofer L. Advances in understanding the complex mechanisms of DNA interstrand cross-link repair. (2013) Cold Spring Harb Perspect Biol. ;5(10):a012732. PubMed PMID: 24086043; PubMed Central PMCID: PMC4123742.

Karakasilioti I, Kamileri I, Chatzinikolaou G, Kosteas T, Vergadi E, Robinson AR, Tsamardinos I, Rozgaja TA, Siakouli S, Tsatsanis C, Niedernhofer LJ, Garinis GA. DNA damage triggers a chronic autoinflammatory response, leading to fat depletion in NER progeria. (2013) Cell Metab. 18(3):403-15. PubMed PMID: 24011075; PubMed Central PMCID: PMC4116679.

Roh DS, Du Y, Gabriele ML, Robinson AR, Niedernhofer LJ, Funderburgh JL. Age-related dystrophic changes in corneal endothelium from DNA repair-deficient mice. (2013) Aging Cell. 12(6):1122-31. PubMed PMID: 23927039; PubMed Central PMCID: PMC4116745.

Cho, J.S., Kook, S.H., Robinson, A.R., Niedernhofer, L.J.* and B.C. Lee*. (2012) Cell autonomous and Non-Autonomous Mechanisms Drive Hematopoietic stem/progenitor Cell Loss in the Absence of DNA Repair. Stem cells, 31(3):511-25. PMC3582850. *co-corresponding authors.

Gregg, S.Q., Gutierrez, V., Robinson, A.R., Woodell, T., Nakao, A., Ross, M.A., Michalopolous, G.K., Rigatti, L., Rothermel, C.E., Kamileri, I., Garinis, G.A., Beer Stolz, D., and L.J. Niedernhofer (2012) A mouse model of accelerated liver aging caused by a defect in DNA repair. Hepatology, 55(2):609-21. PMC3250572

Lavasani, M., Robinson, A.R., Lu, A., Song, M., Feduska, J.M., Ahani, B., Tilstra, J.S., Feldman, C.H., Robbins, P.D., Niedernhofer, L.J.*, J. Huard* (2012) Muscle-derived stem cell/progenitor cell dysfunction limits healthspan and lifespan in a murine progeria model. Nat Commun., 3:608. PMC3272577 *co-corresponding authors.

Tilstra, J.S.*, Robinson, A.R.*, Wang, J.*, Gregg, S.Q.*, Clauson, C.L.*, Reay, D.P., Nasto, L.A., St. Croix, C.M., Usas, A., Vo, N., Huard, J., Clemens, P.R., Stoltz, D.B., Gutteridge, D.C., Watkins, S.C., Garinis, G.A., Wang, Y., Niedernhofer, L.J.*#, and P.D. Robbins# (2012) NF-kB inhibition delays DNA damage-induced senescence and aging in mice. J Clin Invest., 122(7):2601-12. *equal contributors; #co-corresponding authors. PMC3386805.

Wang, J., Clauson, C.L., Robbins, P.D., Niedernhofer, L.J.* and Y. Wang* (2012) The oxidative DNA lesions 8,5'-cyclopurines accumulate with aging in a tissue-specific manner. Aging Cell. 11(4):714-6. PMC3399950 *co-corresponding authors

Vaezi, A., Wang, X-Z, Buch, S., Gooding, W., Wang, L., Seethala, R.R., Weaver, D.T., D'Andrea, A., Argiris, A., Romkes, M., Niedernhofer, L.J.* and J.R. Grandis* (2011) XPF expression correlates with clinical outcome in squamous cell carcinoma of the head and neck. Clin Cancer Res., 17(16):5513-22. PMC3156890 *co-corresponding authors.

Bhagwat, N.R., Olsen, A.L., Wang A.T., Hanada, K., Stuckert, P., Kanaar, R, D'Andrea, A., Niedernhofer, L.J.* and P.J. McHugh* (2009) XPF-ERCC1 participates in the Fanconi anemia pathway of crosslink repair. Mol Cell Biol., 29(24):6427-37. PMC2786876 *co-corresponding authors

Ahmad, A., Robinson, A.R., Duensing, A., van Drunen, E., Beverloo, H.B., Weisberg, D.B., Hasty, P., Hoeijmakers, J.H.J. and L.J. Niedernhofer (2008) ERCC1-XPF endonuclease facilitates DNA double-strand break repair. Mol Cell Biol, 28(16):5082-92. PMC2519706

Niedernhofer, L.J., Garinis, G.A., Raams, A., Lalai, A.S., Robinson, A.R., Appeldoorn, E., Lalai, A., Odijk, H., Oostendorp, R., Ahmad, A., van Leeuwen, W., Theil, A.F., van der Horst, G.T.J., Vermeulen, W., Meinecke, P., Kleijer, W.J., Vijg, J., Jaspers, N.G.J., and J.H.J. Hoeijmakers (2006) A new progeroid syndrome reveals that genotoxic stress suppresses the somatotroph axis. Nature, 444(7122):1038-43.

Niedernhofer, L.J., Odijk, H., Budzowska, M., van Drunen, E., Maas, A., Theil, A.F., de Wit, J., Jaspers, N.G.J., Beverloo, H.B., Hoeijmakers, J.H.J. and Kanaar, R. (2004) The structure-specific endonuclease Ercc1-Xpf is required to resolve DNA interstrand cross-link-induced double-strand breaks. Mol Cell Biol, 24(13):5776-87.

Zhu, X.-D., Niedernhofer, L.J., Kuster, B., Mann, M., Hoeijmakers, J.H.J. and de Lange, T. (2003) ERCC1/XPF removes the 3' overhang from uncapped telomeres and represses formation of telomeric DNA containing double minute chromosomes. Mol Cell, 12(6):1489-98.


Department of Molecular Medicine

Two Noted Scientists Appointed to Metabolism and Aging Department

Molecular Medicine Seminar Series
Center for Physiological Proteomics
Department Contacts

CA Faculty

Asahara, Hiroshi
Balch, William E.
Boddy, Michael
Buxbaum, Joel
Catz, Sergio
Conti, Bruno
Cravatt, Benjamin
D'Lima, Darryl
DerMardirossian, Celine
Deryugina, Elena
Elias, Darlene
Encalada, Sandra
Felding, Brunhilde
Fowler, Velia
Friedlander, Martin
Gerace, Larry
Gottesfeld, Joel
Griffin, John
Hanneken, Anne
Heeb, Mary Jo
Henderson, Scott
Hoch, James
Johnson, Eric
Kanaji, Taisuke
Koziol, James
Lamia, Katja
Lipton, Stuart
Loring, Jeanne F.
Loskutoff, David
Lotz, Martin
Makarenkova, Helen P.
Mc Millan, Robert
Miles, Lindsey
Milner, Richard
Modena, Brian
Mosnier, Laurent
Nicholson, Laura
Paulson, James C.
Perego, Marta
Petrascheck, Michael
Pollard, Kenneth Michael
Quigley, James P.
Reed, Steven
Roberts, Edward
Rosen, Hugh
Ruggeri, Zaverio
Russell, Paul
Saez, Enrique
Smider, Vaughn
Srinivasan, Supriya
Steinhubl, Steven
Sutcliffe, J.
Tan, Eng
Topol, Eric
Vanderklish, Peter
Vogt, Peter K.
Wiseman, Luke
Wittenberg, Curt
Wolan, Dennis W.
Wu, Peng
Wu, Xiaohua
Yang, Xiang-Lei
Yates III, John

FL Faculty

Bannister, Thomas
Bohn, Laura M.
Cameron, Michael
Duckett, Derek
Gill, Matt
Griffin, Patrick
Hansen, Scott
Joazeiro, Claudio
Kamenecka, Theodore
Kissil, Joseph
Luo, Junli
McDonald, Patricia
Miller, Courtney
Niedernhofer, Laura
Phinney, Donald G.
Robbins, Paul
Scampavia, Louis
Schimmel, Paul
Smith, Roy
Spicer, Timothy

Affiliated Faculty

Cline, Hollis
Kojetin, Douglas
Lairson, Luke
Paegel, Brian
Rumbaugh, Gavin
Shen, Ben
Solt, Laura A
Su, Andrew
Wu, Chunlei
Ye, Li

Adjunct Faculty