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Nucleic Acid Therapeutics|February 7, 2025
BMN 351-Induced Exon Skipping and Dystrophin Expression in Skeletal and Cardiac Muscle Lead to Preservation of Motor Function in a Mouse Model of Exon 51 Skip-Amenable Duchenne Muscular DystrophyTodd Oppeneer, Yulan Qi, Joshua Henshaw, et al.
Journal of Molecular and Cellular Cardiology|June 8, 2011
Bi-modal dose-dependent cardiac response to tetrahydrobiopterin in pressure-overload induced hypertrophy and heart failureAn L Moens, Elizabeth A Ketner, Eiki Takimoto, et al.
Clinical Science (London, England : 1979)|March 27, 2010
Tetrahydrobiopterin supplementation reduces atherosclerosis and vascular inflammation in apolipoprotein E-knockout miceTim S Schmidt, Eileen McNeill, Gillian Douglas, et al.
Toxicology and Applied Pharmacology|March 20, 2014
Recombinant human tripeptidyl peptidase-1 infusion to the monkey CNS: safety, pharmacokinetics, and distributionBrian R Vuillemenot, Derek Kennedy, Randall P Reed, et al.
Archives of Ophthalmology (Chicago, Ill. : 1960)|March 15, 2002
Prevention of experimental choroidal neovascularization with intravitreal anti-vascular endothelial growth factor antibody fragmentMagdalena G Krzystolik, Mehran A Afshari, Anthony P Adamis, et al.
Molecular Therapy. Methods & Clinical Development|June 14, 2019
The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene TherapyBrian R Long, Krystal Sandza, Jennifer Holcomb, et al.
Journal of Immunological Methods|October 30, 2016
Antibodies that neutralize cellular uptake of elosulfase alfa are not associated with reduced efficacy or pharmacodynamic effect in individuals with Morquio A syndromeAndrew C Melton, Russell K Soon, Troy Tompkins, et al.
Journal of Neuroscience Research|June 19, 2014
Enzyme replacement therapy attenuates disease progression in a canine model of late-infantile neuronal ceroid lipofuscinosis (CLN2 disease)Martin L Katz, Joan R Coates, Christine M Sibigtroth, et al.
Investigative Ophthalmology & Visual Science|December 31, 2005
Effect of intravitreal injection of ranibizumab in combination with verteporfin PDT on normal primate retina and choroidIvana K Kim, Deeba Husain, Norman Michaud, et al.
Molecular Therapy. Methods & Clinical Development|January 17, 2022
Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate liversChoong-Ryoul Sihn, Britta Handyside, Su Liu, et al.
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