498. Prolonged Benefit from Systemic rAAV8 in a Canine Model of Myotubular Myopathy
Matthew Elverman(University of Washington), Martin K. Childers(University of Washington), David L. Mack(California Institute for Regenerative Medicine), Robert W. Grange(Virginia Tech), Michael W. Lawlor(Medical College of Wisconsin), Virginie Latournerie(Inserm), Karine Poulard(Genethon (France)), Laurine Buscara(Inserm), Hui Meng(Medical College of Wisconsin), Alan H. Beggs(Boston Children's Hospital), Jessica M. Snyder(University of Washington), Melissa Goddard(Forest Institute), P. Véron(Genethon (France)), Samia Martin(Genethon (France)), Federico Mingozzi(Spark Therapeutics (United States)), Anna Buj‐Bello(Pediatrics and Genetics), Christine Le Bec(Genethon (France)), Fulvio Mavilio(The San Raffaele Telethon Institute for Gene Therapy)
Cited by 0
Related Papers
Genotype-Phenotype Correlation in the Long-QT Syndrome
|Circulation|2001|1.8k
Proteomic Analysis of Dendritic Cell-Derived Exosomes: A Secreted Subcellular Compartment Distinct from Apoptotic Vesicles
|The Journal of Immunology|2001|1.5k
Mutations in ACTN4, encoding α-actinin-4, cause familial focal segmental glomerulosclerosis
|Nature Genetics|2000|1.3k
The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion.
|PubMed|1989|963
ACTN3 Genotype Is Associated with Human Elite Athletic Performance
|The American Journal of Human Genetics|2003|917