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Nature Communications|August 29, 2018
Ribitol restores functionally glycosylated α-dystroglycan and improves muscle function in dystrophic FKRP-mutant miceMarcela P Cataldi, Peijuan Lu, Anthony Blaeser, et al.
Molecular Therapy. Methods & Clinical Development|January 29, 2020
ISPD Overexpression Enhances Ribitol-Induced Glycosylation of α-Dystroglycan in Dystrophic FKRP Mutant MiceMarcela P Cataldi, Anthony Blaeser, Peijuan Lu, et al.
Journal of Neuromuscular Diseases|January 26, 2024
Break Down of the Complexity and Inconsistency Between Levels of Matriglycan and Disease Phenotype in FKRP-Related Dystroglycanopathies: A Review and Model of InterpretationQi L Lu, Molly C Holbrook, Marcela P Cataldi, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 3, 2023
Improved efficacy of FKRP AAV gene therapy by combination with ribitol treatment for LGMD2IMarcela P Cataldi, Charles H Vannoy, Anthony Blaeser, et al.
Plos One|January 11, 2018
Distinct expression of functionally glycosylated alpha-dystroglycan in muscle and non-muscle tissues of FKRP mutant miceAnthony Blaeser, Hiroyuki Awano, Pei Lu, et al.
Plos One|October 7, 2016
Progressive Dystrophic Pathology in Diaphragm and Impairment of Cardiac Function in FKRP P448L Mutant MiceAnthony Blaeser, Hiroyuki Awano, Bo Wu, et al.
Molecular Therapy. Methods & Clinical Development|May 9, 2017
Efficacy of Gene Therapy Is Dependent on Disease Progression in Dystrophic Mice with Mutations in the FKRP GeneCharles Harvey Vannoy, Will Xiao, Peijuan Lu, et al.
Human Genetics|April 18, 2013
Mouse models of fukutin-related protein mutations show a wide range of disease phenotypesAnthony Blaeser, Elizabeth Keramaris, Yiumo M Chan, et al.
Human Gene Therapy|January 11, 2014
Evaluation of Tris[2-(acryloyloxy)ethyl]isocyanurate cross-linked polyethylenimine as antisense morpholino oligomer delivery vehicle in cell culture and dystrophic mdx miceMingxing Wang, Bo Wu, Jay D Tucker, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|March 12, 2009
Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx miceBo Wu, Yongfu Li, Paul A Morcos, et al.
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