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Human Gene Therapy|July 9, 2026
AAV.hBAG3 Gene Therapy Improves Phenotype in a Valosin Containing Protein Mouse Model of Hereditary Inclusion Body MyositisBurcak Ozes, Lingying Tong, Morgan Myers, et al.
Neurobiology of Disease|April 24, 2012
Mutant HSPB1 overexpression in neurons is sufficient to cause age-related motor neuronopathy in miceAmit K Srivastava, Samantha R Renusch, Nicole E Naiman, et al.
Experimental Neurology|June 18, 2010
TrkB and TrkC agonist antibodies improve function, electrophysiologic and pathologic features in Trembler J miceZarife Sahenk, Gloria Galloway, Chris Edwards, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience|November 25, 2003
Pathogenesis of X-linked Charcot-Marie-Tooth disease: differential effects of two mutations in connexin 32Charles K Abrams, Mona Freidin, Feliksas Bukauskas, et al.
Neuromuscular Disorders : NMD|June 28, 2025
Like father, like son: RNA-sequencing from a 30-year-old muscle biopsy identifies a novel splice variant in ACTA1 as the cause of an attenuated nemaline myopathy phenotypeAlayne P Meyer, Sana Yousfi, Stefan Nicolau, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 29, 2013
AAV1.NT-3 gene therapy for charcot-marie-tooth neuropathyZarife Sahenk, Gloria Galloway, Kelly Reed Clark, et al.
Journal of Neuropathology and Experimental Neurology|October 17, 2013
Pathogenesis of autosomal dominant hereditary spastic paraplegia (SPG6) revealed by a rat modelFumihiro Watanabe, William D Arnold, Robert E Hammer, et al.
Muscle & Nerve|June 15, 2010
Novel diagnostic features of dysferlinopathiesXiomara Q Rosales, Julie M Gastier-Foster, Sarah Lewis, et al.
Gene Therapy|February 5, 2021
AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1Burcak Ozes, Morgan Myers, Kyle Moss, et al.
Skeletal Muscle|December 16, 2017
Impaired regeneration in calpain-3 null muscle is associated with perturbations in mTORC1 signaling and defective mitochondrial biogenesisMehmet E Yalvac, Jakkrit Amornvit, Cilwyn Braganza, et al.
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