Zarife Sahenk, M.D., Ph.D.
Nationwide Children's Research Institute
Center for Gene Therapy, WA 3024
700 Children's Drive
Columbus, OH 43205
Fax: (614) 355-5247
Education & Training:
Hacettepe University, Ankara, Turkey 1967 B.S.
Ankara, Turkey 1972 M.D.
Ohio State University, 1975 Residency in Neurology
Ohio State University,
1976 Clinical Neuromuscular Fellow
Ohio State University, 1977-78 Neuromuscular
Ohio State University, 1998 Ph.D.
Dr. Sahenk currently serves as Director of Clinical and Experimental
Neuromuscular Pathology at The Research Institute at Nationwide Childrens
Hospital and holds Professorships in Neurology and Pediatrics at the Ohio
State University. Dr. Sahenk’s laboratory focuses on basic and
translational studies related to peripheral nerve disorders. An important
goal has been testing therapeutic options to alleviate nerve dysfunction
in experimental studies in order to contribute pre-clinical data in support
of clinical trials.
Current research aims are to define the anatomical and
molecular basis of hereditary peripheral neuropathies, emphasizing the
role of trophic factors in nerve regeneration and regeneration-associated
myelination. The role of neurotrophin-3 as well as Trk receptor specific
agonistic antibodies in nerve regeneration in animal models of Charcot-Marie-Tooth
(CMT) is currently under investigation. In addition, the laboratory works
on projects involving the development of therapeutics and novel methods
to deliver therapeutic genes more efficiently to the peripheral nerves.
These studies are relevant to understanding the mechanism of axonal loss
and impaired regeneration in CMT disorders and developing rational therapies
to alleviate nerve dysfunction and promote nerve regeneration.
- Sahenk Z, Galloway G, Edwards C, Malik V, Kaspar BK, Eagle A, Yetter B, Forgie A, Tsao D, Lin JC. TrkB and TrkC agonist antibodies improve function, electrophysiologic and pathologic features in Trembler J mice. Experimental neurology. 2010;224:495-506.
- Sahenk Z, Mendell JR. The muscular dystrophies: distinct pathogenic mechanisms invite novel therapeutic approaches. Current rheumatology reports. 2011;13:199-207.
- Srivastava AK, Renusch SR, Naiman NE, Gu S, Sneh A, Arnold WD, Sahenk Z, Kolb SJ. Mutant HSPB1 overexpression in neurons is sufficient to cause age-related motor neuronopathy in mice. Neurobiology of disease. 2012;47:163-73.
- Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Kota J, Malik V, Shontz K, Walker CM, Flanigan KM, Corridore M, Kean JR, Allen HD, Shilling C, Melia KR, Sazani P, Saoud JB, Kaye EM. Eteplirsen for the treatment of Duchenne muscular dystrophy. Annals of neurology. 2013;74:637-47.
- Rodino-Klapac LR, Mendell JR, Sahenk Z. Update on the treatment of Duchenne muscular dystrophy. Current neurology and neuroscience reports. 2013;13:332.
- Rosales XQ, Malik V, Sneh A, Chen L, Lewis S, Kota J, Gastier-Foster JM, Astbury C, Pyatt R, Reshmi S, Rodino-Klapac LR, Clark KR, Mendell JR, Sahenk Z. Impaired regeneration in LGMD2A supported by increased PAX7-positive satellite cell content and muscle-specific microrna dysregulation. Muscle & nerve. 2013;47:731-9.
- Watanabe F, Arnold WD, Hammer RE, Ghodsizadeh O, Moti H, Schumer M, Hashmi A, Hernandez A, Sneh A, Sahenk Z, Kisanuki YY. Pathogenesis of autosomal dominant hereditary spastic paraplegia (SPG6) revealed by a rat model. Journal of neuropathology and experimental neurology. 2013;72:1016-28.
- Sahenk Z, Galloway G, Clark KR, Malik V, Rodino-Klapac LR, Kaspar BK, Chen L, Braganza C, Montgomery C, Mendell JR. AAV1.NT-3 gene therapy for charcot-marie-tooth neuropathy. Molecular therapy : the journal of the American Society of Gene Therapy. 2014;22:511-21.
- Yalvac ME, Arnold WD, Hussain SA, Braganza C, Shontz KM, Clark KR, Walker CM, Ubogu EE, Mendell JR, Sahenk Z. VIP-expressing dendritic cells protect against spontaneous autoimmune peripheral polyneuropathy. Molecular therapy : the journal of the American Society of Gene Therapy. 2014;22:1353-63.
- Yalvac ME, Arnold WD, Braganza C, Chen L, Mendell JR, Sahenk Z. AAV1.NT-3 gene therapy attenuates spontaneous autoimmune peripheral polyneuropathy. Gene therapy. 2016;23:95-102.
- Amornvit J, Yalvac ME, Chen L, Sahenk Z. A novel p.T139M mutation in HSPB1 highlighting the phenotypic spectrum in a family. Brain and behavior. 2017;7:e00774.
- Yalvac ME, Amornvit J, Braganza C, Chen L, Hussain SA, Shontz KM, Montgomery CL, Flanigan KM, Lewis S, Sahenk Z. Impaired regeneration in calpain-3 null muscle is associated with perturbations in mTORC1 signaling and defective mitochondrial biogenesis. Skeletal muscle. 2017;7:27.
- Charleston JS, Schnell FJ, Dworzak J, Donoghue C, Lewis S, Chen L, Young GD, Milici AJ, Voss J, DeAlwis U, Wentworth B, Rodino-Klapac LR, Sahenk Z, Frank D, Mendell JR. Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. Neurology. 2018.
- Yalvac ME, Amornvit J, Chen L, Shontz KM, Lewis S, Sahenk Z. AAV1.NT-3 gene therapy increases muscle fiber diameter through activation of mTOR pathway and metabolic remodeling in a CMT mouse model. Gene therapy. 2018;25:129-38.