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Stem Cells and Development|July 21, 2015
The Role of D4Z4-Encoded Proteins in the Osteogenic Differentiation of Mesenchymal Stromal Cells Isolated from Bone MarrowLaurence de la Kethulle de Ryhove, Eugénie Ansseau, Charlotte Nachtegael, et al.
Molecular Genetics & Genomic Medicine|August 7, 2015
Pseudoexon activation increases phenotype severity in a Becker muscular dystrophy patientKane Greer, Kayla Mizzi, Emily Rice, et al.
Journal of Neurology, Neurosurgery, and Psychiatry|July 17, 1999
Novel mutation in the myelin protein zero gene in a family with intermediate hereditary motor and sensory neuropathyF L Mastaglia, K J Nowak, R Stell, et al.
Neuromuscular Disorders : NMD|January 19, 2010
Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trialsLinda J Popplewell, Carl Adkin, Virginia Arechavala-Gomeza, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 7, 2011
Current status of pharmaceutical and genetic therapeutic approaches to treat DMDChristophe Pichavant, Annemieke Aartsma-Rus, Paula R Clemens, et al.
Movement Disorders : Official Journal of the Movement Disorder Society|February 19, 2009
Do polymorphisms in the familial Parkinsonism genes contribute to risk for sporadic Parkinson's disease?Greg T Sutherland, Glenda M Halliday, Peter A Silburn, et al.
Frontiers in Neuroscience|February 22, 2020
Structural Variants May Be a Source of Missing Heritability in sALSFrances Theunissen, Loren L Flynn, Ryan S Anderton, et al.
Human Gene Therapy|September 5, 2007
Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscleV Arechavala-Gomeza, I R Graham, L J Popplewell, et al.
Plos One|October 23, 2010
Fibulin-1 is increased in asthma--a novel mediator of airway remodeling?Justine Y Lau, Brian G Oliver, Melissa Baraket, et al.
Stem Cell Research|July 1, 2021
Generation of two induced pluripotent stem cell lines from a patient with Stargardt disease caused by compound heterozygous mutations in the ABCA4 geneDi Huang, Dan Zhang, Shang-Chih Chen, et al.
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