Darin Falk, Ph.D.

Darin FalkDivision

Child Health Research Institute

Academic Title

Assistant Professor

Contact Information




  • Postdoctoral Training, University of Florida, College of Medicine
  • Ph.D., University of Florida
  • B.S., University of Nebraska-Kearney


Dr. Falk received his PhD training under Scott K. Powers, PhD, EdD, in the Department of Applied Physiology and Kinesiology at the University of Florida (UF). He continued his training at UF as a NIH postdoctoral fellow in the Department of Pediatrics with Barry J. Byrne, MD, PhD. He has authored over 30 original articles, chapters, and editorials and recently was awarded a Development Grant by the Muscular Dystrophy Association. Dr. Falk also serves as a co-investigator on grants from the NIH and Department of Defense.

Research Overview

The major interest of the laboratory is to determine the primary mechanism by which respiratory muscles display decreased activation or force-generating capacity in models of neuromuscular disease. A specific example is Pompe disease which affects the central nervous system, neuromuscular junction, and skeletal muscle; ultimately resulting in respiratory and ambulatory dysfunction. Our studies have focused on identification of each of these aspects to determine their contribution to loss of proper maintenance and function in these systems. To accomplish this, our approach incorporates an array of molecular, biochemical, and physiological measurements.  In addition, we have found that recombinant adeno-associated viral (rAAV) vectors can be tailored to provide adequate gene expression in skeletal muscle and the CNS in a mouse model of Pompe disease. Specifically, rAAV therapy has reversed the disease associated phenotype and restored motoneuron and respiratory muscle function. Further optimization of rAAV vectors has led to improved transduction efficiency and may allow for a reduction in viral particles required to provide a therapeutic benefit. This may be critical in reducing potential immunoreactivity to the rAAV capsid and/or transgene and provide improved outcomes in affected patients.

Key Publications

Additional publications can be found in PubMed.

  1. Falk DJ, Todd AG, Lee S, Soustek MS, Fuller DD, Notterpek L, Byrne BJ. Peripheral nerve and neuromuscular junction pathology in Pompe disease. Hum Mol Genet. 2014. Accepted with revisions.
  2. Nicks J, Lee S, Harris A, Falk DJ, Todd AG, Arredondo K, Dunn WA Jr, Notterpek L. Rapamycin improves peripheral nerve myelination while it fails to benefit neuromuscular performance in neuropathic mice. Neurobiol Dis. 2014 Jul 9. pii: S0969-9961(14)00187-9. doi: 10.1016/j.nbd.2014.06.023. [Epub ahead of print] PMID: 25014022 [PubMed – as supplied by publisher]
  3. Elmallah MK, Falk DJ, Nayak S, Federico RA, Sandhu MS, Poirier A, Byrne BJ, Fuller DD. Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice. Mol Ther. 2014 Apr;22(4):702-12. doi: 10.1038/mt.2013.282. Epub 2013 Dec 12. PMID: 24336173 [PubMed – in process]
  4. Fuller DD, ElMallah MK, Smith BK, Corti M, Lawson LA, Falk DJ, Byrne BJ. The respiratory neuromuscular system in Pompe disease. Respir Physiol Neurobiol. 2013 Nov 1;189(2):241-9. doi: 10.1016/j.resp.2013.06.007. Epub 2013 Jun 21. Review. PMID: 23797185 [PubMed – indexed for MEDLINE]
  5. Falk DJ, Mah CS, Soustek MS, Lee KZ, Elmallah MK, Cloutier DA, Fuller DD, Byrne BJ. Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease. Mol Ther. 2013 Sep;21(9):1661-7. doi: 10.1038/mt.2013.96. Epub 2013 Jun 4. PMID: 23732990 [PubMed – indexed for MEDLINE]