H. Lee Sweeney is an American scientist who studies

muscle Muscle is a soft tissue, one of the four basic types of animal tissue. There are three types of muscle tissue in vertebrates: skeletal muscle, cardiac muscle, and smooth muscle. Muscle tissue gives skeletal muscles the ability to muscle contra ...

Education and career

He received his undergraduate degree in biochemistry from

Massachusetts Institute of Technology The Massachusetts Institute of Technology (MIT) is a Private university, private research university in Cambridge, Massachusetts, United States. Established in 1861, MIT has played a significant role in the development of many areas of moder ...

in 1975 and his PhD from

Harvard Harvard University is a private Ivy League research university in Cambridge, Massachusetts, United States. Founded in 1636 and named for its first benefactor, the Puritan clergyman John Harvard, it is the oldest institution of higher lear ...

in physiology and

biophysics Biophysics is an interdisciplinary science that applies approaches and methods traditionally used in physics to study biological phenomena. Biophysics covers all scales of biological organization, from molecular to organismic and populations ...

in 1984. 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 The University of Texas at Austin (UT Austin, UT, or Texas) is a public university, public research university in Austin, Texas, United States. Founded in 1883, it is the flagship institution of the University of Texas System. With 53,082 stud ...

. He joined the faculty of the

University of Pennsylvania The University of Pennsylvania (Penn or UPenn) is a Private university, private Ivy League research university in Philadelphia, Pennsylvania, United States. One of nine colonial colleges, it was chartered in 1755 through the efforts of f ...

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. He left Penn and joined the faculty of

University of Florida College of Medicine The University of Florida College of Medicine is the medical school of the University of Florida. It is part of the J. Hillis Miller Health Science Center, with facilities in Gainesville and Jacksonville, Florida. The school grants Doctor ...

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.

Research

In 1998 his lab published work in which they used

gene therapy Gene therapy is Health technology, medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells. The first attempt at modifying human DNA ...

to incorporate an extra copy of

IGF1 Insulin-like growth factor 1 (IGF-1), also called somatomedin C, is a hormone similar in molecular structure to insulin which plays an important role in childhood growth, and has anabolic effects in adults. In the 1950s IGF-1 was called " sulfa ...

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 Gene doping is the hypothetical non-therapeutic use of gene therapy by athletes in order to improve their performance in those sporting events which prohibit such applications of genetic modification technology, and for reasons other than the tre ...

; he was still receiving inquires as of 2008. He has written on the topic of gene doping in popular media and is often called upon to comment on the topic in the media. Sweeney was awarded Hamdan Award for Medical Research Excellence - Molecular Therapy in Drug Targeting (Pharmacogenomics) in 2008 by Hamdan Medical Award. Sweeney collaborated with

PTC Therapeutics PTC Therapeutics, Inc. is a US pharmaceutical company focused on the development of orally administered small molecule drugs and gene therapy which regulate gene expression by targeting post-transcriptional control (PTC) mechanisms in orphan ...

in the discovery and development of

ataluren Ataluren, sold under the brand name Translarna, is a medication for the treatment of Duchenne muscular dystrophy. It was designed by PTC Therapeutics. Medical use Ataluren is used in the European Union to treat people with Duchenne muscular dy ...

. which was initially funded in part by Parent Project Muscular Dystrophy. Sweeney's lab had published work in 1999 showing that

gentamicin Gentamicin is an aminoglycoside antibiotic used to treat several types of bacterial infections. This may include bone infections, endocarditis, pelvic inflammatory disease, meningitis, pneumonia, urinary tract infections, and sepsis amo ...

could "cure" a rodent model of

Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy predominantly affecting boys. The onset of muscle weakness typically begins around age four, with rapid progression. Initially, muscle loss occurs in the thighs and pe ...

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. Ataluren was approved to treat DMD in Europe in 2014. As of 2012 he had been an author on around 180 papers and reviews that had been cited around 16,000 times. Along with serving on the scientific advisory board of PTC, he does likewise with

Cytokinetics Cytokinetics, Incorporated, is a biopharmaceutical company based in South San Francisco, California, that develops muscle activators and muscle inhibitors as potential treatments for people with diseases characterized by impaired or declining mus ...

and Solid Biosciences He is also on the scientific advisory board of the Parent Project Muscular Dystrophy.

References

{{DEFAULTSORT:Sweeney, Lee American physiologists Harvard University alumni University of Florida faculty Living people Massachusetts Institute of Technology School of Science alumni Year of birth missing (living people)