Department of Neurology


Selected Peer-Reviewed Publications

Listed here are the most relevant to our current research interests.

  • Odom GL, Gregorevic P, Allen JM, Finn EE, Chamberlain JS. Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice. Mol Ther. 2008 Sep;16(9):1539-45. (PMID: 18665159)
  • Odom GL, Banks GB, Schultz BR, Gregorevic P, Chamberlain JS. Preclinical studies for gene therapy of Duchenne muscular dystrophy. J. Child Neurol. 2010 Sep; 25(9):1149-57. (PMID: 20498332)
  • Odom GL, Gregorevic P, Allen JM, Chamberlain JS. Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6. Mol Ther. 2011;19(1): 36-45. (PMID: 20859263)
  • Bieber S, Halldorson JB, Finn E, Chamberlain JS, and Odom GL. Extracorporeal Delivery of rAAV with Metabolic Exchange and Oxygenation. Nature Scientific Reports. 2013;3:1538. (PMID: 23528884)
  • Banks GB, Combs A, Odom GL, and Chamberlain JS. Muscle structure influences utrophin expression in mdx mice. PLOS Genetics, 2014; Jun 12;10(6). (PMID: 24922526)
  • Arnett A, Konieczny P, Ramos JN, Hall J, Odom GL, Yablonka-Reuveni Z, Chamberlain JR and Chamberlain JS. Adeno-associated viral vectors do not efficiently target muscle satellite cells. Mol. Ther. Meth. & Clin. Dev. Sept. 2014, 1;14038. (PMID: 25580445)
  • Strakova J, Dean JD, Sharpe K, Meyers TA, Odom GL, and Townsend D. Alpha-dystrobrevin potentiates the function of dystrophin function in the heart by reinforcing dystrophin’s interaction with the membrane. J. Mol. Cell Cardiol. 2014 Nov. 76:106-115. (PMID: 25158611)
  • Swiderski K, Shaffer S, Gallis B, Odom GL, Arnett AL, Edgar JS, Baum DM, Chee A, Naim T, Gregorevic, P, Murphy, KT, Goodlett, DR, Lynch GS, and Chamberlain, JS. Phosphorylation within the cysteine-rich region of dystrophin enhances its association with β-dystroglycan and identifies a potential novel therapeutic target for skeletal muscle wasting. Hum Mol Genet. 2014 Dec 20; 23(25):6697-711. (PMID: 25082828)
  • Su W, Kang J, Sopher B, Gillespie J, Macarena, AS, Odom GL, Case A, Wang DB, Chamberlain JS, and Garden G. Adeno-associated virus (AAV) vectors mediate efficient gene transduction in cultured neonatal and adult microglia. J. Neurochem. 2016 Jan; 136 Suppl 1:49-62. (PMID: 25708596)
  • Odom GL, Kolwicz SC, Nowakowski SG, Moussavi-Harami, F, Flint GV, Gay E, Stuppard R, Gu H, Djukovic D, Chamberlain JS, Raftery D, Tian R, Marcinek DJ, Murry C, and Regnier M. AAV6-mediated Cardiac Specific Over-expression of Ribonucleotide Reductase Enhances Myocardial Contractility. Mol. Ther. 2016 Feb; 24(2):240-50. (PMID: 26388461)
  • Whitehead N, Bible K, Kim MJ, Adams M, Odom, GL and Froehner SC. Validation of ultrasonography for non-invasive assessment of diaphragm function in muscular dystrophy. J Physiol. 2016 Dec 15;594(24):7215-27. (PMID: 27570057)
  • Bengtsson N, Hall J, Odom G, Phelps MP, Andrus CR, Hawkins DR, Hauschka SD, Chamberlain JC, and Chamberlain JS. Enhanced muscle-specific CRISPR/Cas9 editing of the dystrophin gene ameliorates pathophysiology in a mouse model of DMD. Nature Comm. 2017 Feb; 8:14454.
  • Adams ME, Odom GL, Kim MJ, Chamberlain JS, and Froehner SC.  Syntrophin binds directly to multiple spectrin-like repeats in dystrophin and mediates binding of nNOS to repeats 16-17.  Human Molecular Genetics 2018 May 22.  (PMID: 29790927)
  • Kennedy TL, Guiraud S, Edwards B, Squire S, Moir L, Babbs A, Odom GL Chamberlain, JS, and Davies KE.  Micro-utrophin improves cardiac and skeletal muscle function of severely affected D2/mdx mice. Mol. Therapy Methods Clin Dev. 2018 Oct 16;11:92-105.  (PMID: 30417024)
  • Kolwicz SC, Hall JK, Moussavi-Harami F, Hauschka SD, Chamberlain JS, Regnier M, and Odom GL.  Elevating cardiac deoxy-ATP via gene therapy rescues cardiac dysfunction in mice with Duchenne muscular dystrophy.   JACC-basic to translational science, Oct 2:4(7):778-791, 2019.  (PMID: 31998848)
  • Banks GB, Chamberlain JS, and Odom GL.  Microutrophin expression in dystrophic mice displays myofiber type differences in therapeutic effects.  PLOS Genetics, Nov 16(11):e1009179 epub, 2020.  (PMID: 33175853)
  • Howard ZM, Dorn LE, Lowe J, Gertzen MD, Ciccone P, Rastogi N, Odom GL, Accornero F, Chamberlain JS, and Rafael-Fortney JA.  Micro-dystrophin gene therapy prevents heart failure in an improved Duchenne muscular dystrophy cardiomyopathy mouse model. JCI Insight, 2021;6(7):e146511. (PMID: 33651713)


  • Odom GL, Gregorevic P, and Chamberlain JS. Viral-mediated gene therapy for the muscular dystrophies: successes, limitations and recent advances.  Bioch Biophys Acta. 2007, Feb; 1771(2):243-62.  (PMID: 17064882)
  • Seto J, Ramos JN, Muir L, Chamberlain JS, and Odom GL.  Gene replacement therapies for Duchenne muscular dystrophy using adeno-associated viral vectors.  Current Gene Therapy. 2012 Jun 1;12(3):139-51.  (PMID: 22533379)
  • Bengtsson N, Seto JS, Hall JK, Chamberlain JS, and Odom GL. Progress and Prospects of Gene therapy clinical trials for the muscular dystrophies. Hum Mol Genet. 2016 Apr 15;25(R1):R9-R17.  (PMID: 26450518)
  • Thompson KS, Odom GL, Murry CE, Mahairas GG, Moussavi-Harami F, Teichman SL, Chan X, Hauschka SD, Chamberlain JS, Regnier M.  Overexpression of Ribonucleotide Reductase with Gene Therapy Treats Heart Failure by Activating Cardiac Myosin and Enhancing Myocardial Contractility.  JACC 2016 Dec 1; 1(7):666-679.