Lee Sweeney

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H. Lee Sweeney is an American scientist who studies muscle.

Education and career[edit]

He received his undergraduate degree in biochemistry from Massachusetts Institute of Technology in 1975 and his PhD from Harvard in physiology and biophysics in 1984.[1] He then spent a year as research instructor in physiology at the University of Texas Southwestern Medical School, and then obtained an appointment as an assistant professor at the University of Texas at Austin.[2]

He joined the faculty of the University of Pennsylvania in 1989, became chair of the department of physiology in 1999, became founding director of the Paul D. Wellstone Muscular Dystrophy Cooperative Research Center in 2005. and eventually obtained an endowed chair and became director of the Penn Center for Orphan Disease Research and Therapy, which was founded in 2012.[2][3]

He left Penn and joined the faculty of University of Florida College of Medicine in 2015, where he became the Thomas H. Maren M.D. Eminent Scholar Chair in Pharmacology and Therapeutics and became the director of the new Myology Institute there.[3]


In 1998 his lab published work in which they used gene therapy to incorporate an extra copy of IGF1 into muscle cells of young and old mice; age-related muscle aging in the older mice was reversed and both old and mice became stronger. The paper received national press coverage, and the mice became known as "Schwarzenegger mice." He soon started receiving calls from athletes and coaches asking him to use this method for gene doping; he was still receiving inquires as of 2008.[4][5] He has written on the topic of gene doping in popular media[6] and is often called upon to comment on the topic in the media.[7][8][9]

Sweeney collaborated with PTC Therapeutics in the discovery and development of ataluren.[10][11] which was initially funded in part by Parent Project Muscular Dystrophy.[12] Sweeney's lab had published work in 1999 showing that gentamicin could "cure" a rodent model of Duchenne muscular dystrophy[10][13] and this led to the collaboration with PTC that produced ataluren—Sweeney was the last author on the initial publication of ataluren in Nature in 2007.[14] Ataluren was approved to treat DMD in Europe in 2014.[15]

As of 2012 he had been an author on around 180 papers and reviews that had been cited around 16,000 times.[2]

Along with serving on the scientific advisory board of PTC, he does likewise with Cytokinetics[16] and Solid Biosciences[17] He is also on the scientific advisory board of the Parent Project Muscular Dystrophy.[18]


  1. ^ "H. Lee Sweeney, Ph.D". Perelman School of Medicine at the University of Pennsylvania. Archived from the original on 7 July 2015. Retrieved 18 June 2017.
  2. ^ a b c "Press Release: Penn Scientist Named First Director of New Center for Orphan Disease Research and Therapy – PR News". Penn Medicine. November 28, 2012.
  3. ^ a b "UF Recruits Muscle Expert". Health News Florida. May 29, 2014.
  4. ^ Wenner, Melinda (August 15, 2008). "How to Be Popular during the Olympics: Be H. Lee Sweeney, Gene Doping Expert". Scientific American.
  5. ^ Epstein, David (2013). The Sports Gene: Inside the Science of Extraordinary Athletic Performance. Penguin. pp. 77ff. ISBN 9781101622636.
  6. ^ Sweeney, H. Lee (July 2004). "Gene Doping". Scientific American. pp. 62–69. doi:10.1038/scientificamerican0704-62.
  7. ^ Hamilton, Jon (August 12, 2013). "New Muscle Drugs Could Be The Next Big Thing In Sports Doping". Morning Edition, NPR.
  8. ^ Coghlan, Andy (14 March 2012). "Blood tests won't stop gene cheats". New Scientist.
  9. ^ DeLessio, Joe (31 March 2015). "Genetic Doping Is the Next Frontier of Cheating in Sports". New York Magazine.
  10. ^ a b Maugh II, Thomas H. (28 April 2007). "Drug may cure genetic diseases". Los Angeles Times.
  11. ^ "Prof. H Lee Sweeney". Sheikh Hamdan Bin Rashid Al Maktoum Award for Medical Sciences - HMA. 2008. Retrieved 18 June 2017.
  12. ^ "Press release: PTC Therapeutics Announces $15.4 Million NIH Research Grant for Duchenne Muscular Dystrophy | Evaluate". PTC, University of Pennsylvania, and the NIH via Evaluate Group. July 10, 2007.
  13. ^ Barton-Davis, ER; Cordier, L; Shoturma, DI; Leland, SE; Sweeney, HL (August 1999). "Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice". The Journal of Clinical Investigation. 104 (4): 375–81. doi:10.1172/jci7866. PMC 481050. PMID 10449429.
  14. ^ Welch, EM; Barton, ER; Zhuo, J; Tomizawa, Y; Friesen, WJ; Trifillis, P; Paushkin, S; Patel, M; Trotta, CR; Hwang, S; Wilde, RG; Karp, G; Takasugi, J; Chen, G; Jones, S; Ren, H; Moon, YC; Corson, D; Turpoff, AA; Campbell, JA; Conn, MM; Khan, A; Almstead, NG; Hedrick, J; Mollin, A; Risher, N; Weetall, M; Yeh, S; Branstrom, AA; Colacino, JM; Babiak, J; Ju, WD; Hirawat, S; Northcutt, VJ; Miller, LL; Spatrick, P; He, F; Kawana, M; Feng, H; Jacobson, A; Peltz, SW; Sweeney, HL (3 May 2007). "PTC124 targets genetic disorders caused by nonsense mutations". Nature. 447 (7140): 87–91. doi:10.1038/nature05756. PMID 17450125.
  15. ^ "Translarna - Summary of Product Characteristics". UK Electronic Medicines Compendium. 24 April 2017. Retrieved 18 June 2017.
  16. ^ "H. Lee Sweeney, Ph.D." Cytokinetics. Retrieved 18 June 2017.
  17. ^ "Lee Sweeney, Ph.D." Solid Biosciences. 6 April 2016. Retrieved 18 June 2017.
  18. ^ "Scientific Advisory Committee (SAC) - Parent Project Muscular Dystrophy". Parent Project Muscular Dystrophy. Retrieved 18 June 2017.