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Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial Russell, Stephen R.; Drack, Arlene, V; Cideciyan, Artur, V; Jacobson, Samuel G.; Leroy, Bart P.; Van Cauwenbergh, Caroline; Ho, Allen C.; Dumitrescu, Alina, V; Han, Ian C.; Martin, Mitchell; Pfeifer, Wanda L.; Sohn, Elliott H.; Walshire, Jean; Garafalo, Alexandra, V; Krishnan, Arun K.; Powers, Christian A.; Sumaroka, Alexander; Roman, Alejandro J.; Vanhonsebrouck, Eva; Jones, Eltanara; Nerinckx, Fanny; De Zaeytijd, Julie; Collin, Rob W. J.; Hoyng, Carel; Adamson, Peter; Cheetham, Michael E.; Schwartz, Michael R.; den Hollander, Wilhelmina; Asmus, Friedrich; Platenburg, Gerard; Rodman, David; Girach, Aniz 分享 收藏
Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations Dulla, Kalyan; Slijkerman, Ralph; van Diepen, Hester C.; Albert, Silvia; Dona, Margo; Beumer, Wouter; Turunen, Janne J.; Chan, Hee Lam; Schulkens, Iris A.; Vorthoren, Lars; den Besten, Cathaline; Buil, Levi; Schmidt, Iris; Miao, Jiayi; Venselaar, Hanka; Zang, Jingjing; Neuhauss, Stephan C. F.; Peters, Theo; Broekman, Sanne; Pennings, Ronald; Kremer, Hannie; Platenburg, Gerard; Adamson, Peter; de Vrieze, Erik; van Wijk, Erwin 分享 收藏
Antisense oligonucleotide treatment in CEP290-related leber congenital amaurosis Leroy, Bart P.; Cideciyan, Artur V.; Jacobson, Samuel G.; Drack, Arlene V.; Ho, Allen C.; Garafalo, Alexandra; Roman, Alejandro J.; Schwartz, Mike; Biasutto, Patricia; De Wit, Wilma; Cheetham, Michael E.; Adamson, Peter S.; Rodham, David; Girach, Aniz; De Zaeytijd, Julie; Van Cauwenbergh, Caroline; Russell, Stephen R. 分享 收藏
Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect Cideciyan, Artur V.; Jacobson, Samuel G.; Drack, Arlene V.; Ho, Allen C.; Charng, Jason; Garafalo, Alexandra V.; Roman, Alejandro J.; Sumaroka, Alexander; Han, Ian C.; Hochstedler, Maria D.; Pfeifer, Wanda L.; Sohn, Elliott H.; Taiel, Magali; Schwartz, Michael R.; Biasutto, Patricia; de Wit, Wilma; Cheetham, Michael E.; Adamson, Peter; Rodman, David M.; Platenburg, Gerard; Tome, Maria D.; Balikova, Irina; Nerinckx, Fanny; De Zaeytijd, Julie; Van Cauwenbergh, Caroline; Leroy, Bart P.; Russell, Stephen R. 分享 收藏
Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models Dulla, Kalyan; Aguila, Monica; Lane, Amelia; Jovanovic, Katarina; Parfitt, David A.; Schulkens, Iris; Chan, Hee Lam; Schmidt, Iris; Beumer, Wouter; Vorthoren, Lars; Collin, Rob W. J.; Garanto, Alejandro; Duijkers, Lonneke; Brugulat-Panes, Anna; Semo, Ma'ayan; Vugler, Anthony A.; Biasutto, Patricia; Adamson, Peter; Cheetham, Michael E. 分享 收藏
Antisense Therapy for a Common Corneal Dystrophy Ameliorates TCF4 Repeat Expansion-Mediated Toxicity 常见角膜营养不良的反义疗法可改善TCF4重复扩增介导的毒性 Zarouchlioti, Christina; Sanchez-Pintado, Beatriz; Tear, Nathaniel J. Hafford; Klein, Pontus; Liskova, Petra; Dulla, Kalyan; Semo, Ma'ayan; Vugler, Anthony A.; Muthusamy, Kirithika; Dudakova, Lubica; Levis, Hannah J.; Skalicka, Pavlina; Hysi, Pirro; Cheetham, Michael E.; Tuft, Stephen J.; Adamson, Peter; Hardcastle, Alison J.; Davidson, Alice E. 分享 收藏
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Endothelial MAPKs Direct ICAM-1 Signaling to Divergent Inflammatory Functions Dragoni, Silvia; Hudson, Natalie; Kenny, Bridget-Ann; Burgoyne, Thomas; McKenzie, Jenny A.; Gill, Yadvinder; Blaber, Robert; Futter, Clare E.; Adamson, Peter; Greenwood, John; Turowski, Patric 分享 收藏
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Single ocular injection;
a sustained-release anti-VEGF delivers 6 months pharmacokinetics and efficacy in a primate laser CNV model Adamson, Peter; Wilde, Thomas; Dobrzynski, Eric; Sychterz, Caroline; Polskyc, Rodd; Kurali, Edit; Haworth, Richard; Tang, Chi-Man; Korczynska, Justyna; Cook, Fiona; Papanicolaou, Irene; Tsikna, Lemy; Roberts, Chris; Hughes-Thomas, Zoe; Walford, James; Gibson, Daniel; Warrack, John; Smal, Jos; Verrijk, Ruud; Miller, Paul E.; Nork, T. Michael; Prusakiewicz, Jeffery; Streit, Timothy; Sorden, Steven; Struble, Craig; Christian, Brian; Catchpole, Ian R. 分享 收藏
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Lipoprotein-associated phospholipase A2 (Lp-PLA2) as a therapeutic target to prevent retinal vasopermeability during diabetes 脂蛋白相关磷脂酶A2 (Lp-PLA2) 作为预防糖尿病期间视网膜血管可透性的治疗靶标 Canning, Paul; Kenny, Bridget-Ann; Prise, Vivien; Glenn, Josephine; Sarker, Mosharraf H.; Hudson, Natalie; Brandt, Martin; Lopez, Francisco J.; Gale, David; Luthert, Philip J.; Adamson, Peter; Turowski, Patric; Stitt, Alan W. 分享 收藏
T-bet Activates Th1 Genes through Mediator and the Super Elongation Complex Hertweck, Arnulf; Evans, Catherine M.; Eskandarpour, Malihe; Lau, Jonathan C. H.; Oleinika, Kristine; Jackson, Ian; Kelly, Audrey; Ambrose, John; Adamson, Peter; Cousins, David J.; Lavender, Paul; Calder, Virginia L.; Lord, Graham M.; Jenner, Richard G. 分享 收藏
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Patient demographics and cardiovascular risk factors differentially influence geometric remodeling of the aorta compared with the peripheral arteries Kamenskiy, Alexey; Miserlis, Dimitrios; Adamson, Peter; Adamson, Micah; Knowles, Thomas; Neme, Jamil; Koutakis, Panagiotis; Phillips, Nicholas; Pipinos, Iraklis; MacTaggart, Jason 分享 收藏
Allele-Specific Inhibition of Rhodopsin With an Antisense Oligonucleotide Slows Photoreceptor Cell Degeneration Murray, Susan F.; Jazayeri, Ali; Matthes, Michael T.; Yasumura, Douglas; Yang, Haidong; Peralta, Raechel; Watt, Andy; Freier, Sue; Hung, Gene; Adamson, Peter S.; Guo, Shuling; Monia, Brett P.; LaVail, Matthew M.; McCaleb, Michael L. 分享 收藏
IL-18 Immunotherapy for Neovascular AMD: Tolerability and Efficacy in Nonhuman Primates Doyle, Sarah L.; Lopez, Francisco J.; Celkova, Lucia; Brennan, Kiva; Ozaki, Ema; Mulfaul, Kelly; Kenna, Paul F.; Kurali, Edit; Hudson, Natalie; Doggett, Teresa; Ferguson, Thomas A.; Humphries, Peter; Adamson, Peter; Campbell, Matthew 分享 收藏
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