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Topical Application of a Novel Harderian-Derived Nonpolar Lipid Prolongs Tear Film Breakup Time in Normal Beagle Dogs 一种源自哈德腺的新型非极性脂质局部应用可延长正常比格犬的泪膜破裂时间 Lantyer-Araujo, Nayone L.; Park, Sangwan; Hisey, Erin A.; Thomasy, Sara M.; Murphy, Christopher J.; Albert, Daniel M.; O'Neill, Charles A.; Gadek, Thomas R.; Parikh, Atul N.; Leonard, Brian C. 分享 收藏
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 Dystrophy Oppeneer, Todd; Qi, Yulan; Henshaw, Joshua; Larimore, Kevin; Puolivaeli, Jukka; Carter, Caitlyn; Fant, Pierluigi; Brennan, Sebastian; Wetzel, Laura A.; Sigg, Monika A.; O'Neill, Charles A. 分享 收藏
Targeting a Novel Site in Exon 51 with Antisense Oligonucleotides Induces Enhanced Exon Skipping in a Mouse Model of Duchenne Muscular Dystrophy Oppeneer, Todd; Qi, Yulan; Henshaw, Joshua; Larimore, Kevin; Melton, Andrew; Puolivali, Jukka; Carter, Caitlyn; Fant, Pierluigi; Brennan, Sebastian; Wetzel, Laura A.; Sigg, Monika A.; Crawford, Brett E.; Magat, Jenna; Froelich, Steven; Woloszynek, Josh C.; O'Neill, Charles A. 分享 收藏
pathfinder: A Semantic Framework for Literature Review and Knowledge Discovery in Astronomy Iyer, Kartheik G.; Yunus, Mikaeel; O'Neill, Charles; Ye, Christine; Hyk, Alina; Mccormick, Kiera; Ciuca, Ioana; Wu, John F.; Accomazzi, Alberto; Astarita, Simone; Chakrabarty, Rishabh; Cranney, Jesse; Field, Anjalie; Ghosal, Tirthankar; Ginolfi, Michele; Huertas-Company, Marc; Jablonska, Maja; Kruk, Sandor; Liu, Huiling; Marchidan, Gabriel; Mistry, Rohit; Naiman, J. P.; Peek, J. E. G.; Polimera, Mugdha; Mendez, Sergio J. Rodriguez; Schawinski, Kevin; Sharma, Sanjib; Smith, Michael J.; Ting, Yuan-Sen; Walmsley, Mike 分享 收藏
Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy 下一代针对杜氏肌营养不良症的外显子51跳跃反义寡核苷酸 van Deutekom, Judith; Beekman, Chantal; Bijl, Suzanne; Bosgra, Sieto; van den Eijnde, Rani; Franken, Dennis; Groenendaal, Bas; Harquouli, Bouchra; Janson, Anneke; Koevoets, Paul; Mulder, Melissa; Muilwijk, Daan; Peterburgska, Galyna; Querido, Bianca; Testerink, Janwillem; Verheul, Ruurd; de Visser, Peter; Weij, Rudie; Aartsma-Rus, Annemieke; Puolivali, Jukka; Bragge, Timo; O'Neill, Charles; Datson, Nicole A. 分享 收藏
Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB Ellinwood, N. Matthew; Valentine, Bethann N.; Hess, Andrew S.; Jens, Jackie K.; Snella, Elizabeth M.; Jamil, Maryam; Hostetter, Shannon J.; Jeffery, Nicholas D.; Smith, Jodi D.; Millman, Suzanne T.; Parsons, Rebecca L.; Butt, Mark T.; Chandra, Sundeep; Egeland, Martin T.; Assis, Ana B.; Nelvagal, Hemanth R.; Cooper, Jonathan D.; Nestrasil, Igor; Mueller, Bryon A.; Labounek, Rene; Paulson, Amy; Prill, Heather; Liu, Xiao Ying; Zhou, Huiyu; Lawrence, Roger; Crawford, Brett E.; Grover, Anita; Cherala, Ganesh; Melton, Andrew C.; Cherukuri, Anu; Vuillemenot, Brian R.; Wait, Jill C. M.; O'Neill, Charles A.; Pinkstaff, Jason; Kovalchin, Joseph; Zanelli, Eric; McCullagh, Emma 分享 收藏
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Intravitreal enzyme replacement inhibits progression of retinal degeneration in canine CLN2 neuronal ceroid lipofuscinosis Whiting, Rebecca E. H.; Kick, Grace Robinson; Ota-Kuroki, Juri; Lim, Stefanie; Castaner, Leilani J.; Jensen, Cheryl A.; Kowal, Joseph; Nguyen, Annalisa; Corado, Carley; O'Neill, Charles A.; Katz, Martin L. 分享 收藏
Intravitreal enzyme replacement preserves retinal structure and function in canine CLN2 neuronal ceroid lipofuscinosis Whiting, Rebecca E. H.; Pearce, Jacqueline W.; Vansteenkiste, Daniella P.; Bibi, Katherine; Lim, Stefanie; Kick, Grace Robinson; Castaner, Leilani J.; Sinclair, John; Chandra, Sundeep; Nguyen, Annalisa; O'Neill, Charles A.; Katz, Martin L. 分享 收藏
Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B Grover, Anita; Crippen-Harmon, Danielle; Nave, Lacey; Vincelette, Jon; Wait, Jill C. M.; Melton, Andrew C.; Lawrence, Roger; Brown, Jillian R.; Webster, Katherine A.; Yip, Bryan K.; Baridon, Brian; Vitelli, Catherine; Rigney, Sara; Christianson, Terri M.; Tiger, Pascale M. N.; Lo, Melanie J.; Holtzinger, John; Shaywitz, Adam J.; Crawford, Brett E.; Fitzpatrick, Paul A.; LeBowitz, Jonathan H.; Bullens, Sherry; Aoyagi-Scharber, Mika; Bunting, Stuart; O'Neill, Charles A.; Pinkstaff, Jason; Bagri, Anil 分享 收藏
Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice Bunting, Stuart; Zhang, Lening; Xie, Lin; Bullens, Sherry; Mahimkar, Rajeev; Fong, Sylvia; Sandza, Krystal; Harmon, Danielle; Yates, Bridget; Handyside, Britta; Sihn, Choong-Ryoul; Galicia, Nicole; Tsuruda, Laurie; O'Neill, Charles A.; Bagri, Anil; Colosi, Peter; Long, Shinong; Vehar, Gordon; Carter, Barrie 分享 收藏
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Reveglucosidase alfa (BMN 701), an IGF2-Tagged rhAcid α-Glucosidase, Improves Respiratory Functional Parameters in a Murine Model of Pompe Disease Peng, Jeffrey; Dalton, Jill; Butt, Mark; Tracy, Kristin; Kennedy, Derek; Haroldsen, Peter; Cahayag, Rhea; Zoog, Stephen; O'Neill, Charles A.; Tsuruda, Laurie S. 分享 收藏
Immunogenicity to cerliponase alfa, an enzyme replacement therapy for patients with CLN2 disease: results from a phase 1/2 study Cherukuri, Anu; Cahan, Heather; Van Tuyl, Andrea; de Hart, Greg; Slasor, Peter; Bray, Laurie; Henshaw, Josh; Ajayi, Temitayo; Jacoby, Dave; O'Neill, Charles; Schweighardt, Becky 分享 收藏
Long-term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study Long, Brian; Tompkins, Troy; Decker, Celeste; Jesaitis, Lynne; Khan, Shahid; Slasor, Peter; Harmatz, Paul; O'Neill, Charles A.; Schweighardt, Becky 分享 收藏
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Pharmacodynamics, pharmacokinetics and biodistribution of recombinant human N-acetylgalactosamine 4-sulfatase after 6 months of therapy in cats using different IV infusion durations Ruane, Therese; Haskins, Mark; Cheng, Alphonsus; Wang, Ping; Aguirre, Gustavo; Knox, Van W. (Rafe); Qi, Yulan; Tompkins, Troy; O'Neill, Charles A. 分享 收藏
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