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Therapeutic antisense oligonucleotide mitigates retinal dysfunction in a pig model of CLN3 Batten disease 治疗性反义寡核苷酸缓解CLN3巴顿病猪模型中的视网膜功能障碍 Stratton, Matthew P.; Centa, Jessica L.; Swier, Vicki J.; Pfeifer, Wanda L.; Booth, Clarissa D.; Albert, Karlee; Hunyara, John L.; Rechtzigel, Mitchell J.; Duelli, Fox J.; Leppert, Hannah G.; Rigo, Frank; Smit, Trisha; Jafar-nejad, Paymaan; Weimer, Jill M.; Drack, Arlene, V; Hastings, Michelle L. 分享 收藏
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Conjugation to a transferrin receptor 1-binding Bicycle peptide enhances ASO and siRNA potency in skeletal and cardiac muscles 与转铁蛋白受体1结合的Bicycle肽偶联可增强ASO和siRNA在骨骼肌和心肌中的效力 Ostergaard, Michael E.; Carrer, Michele; Anderson, Brooke A.; Afetian, Megan; Bakooshli, Mohsen A.; Santos, Jinro A.; Klein, Stephanie K.; Capitanio, Juliana; Freestone, Graeme C.; Tanowitz, Michael; Galindo-Murillo, Rodrigo; Gaus, Hans J.; Dwyer, Chrissa A.; Jackson, Michaela; Jafar-nejad, Paymaan; Rigo, Frank; Seth, Punit P.; Gaynor, Katherine U.; Stanway, Steven J.; Urbonas, Liudvikas; St. Denis, Megan A.; Pellegrino, Simone; Bezerra, Gustavo A.; Rigby, Michael; Gowans, Ellen; Van Rietschoten, Katerine; Beswick, Paul; Skynner, Michael J.; Swayze, Eric E. 分享 收藏
Modeling antisense oligonucleotide therapy in MECP2 duplication syndrome human iPSC-derived neurons reveals gene expression programs responsive to MeCP2 levels Bajikar, Sameer S.; Sztainberg, Yehezkel; Trostle, Alexander J.; Tirumala, Harini P.; Wan, Ying-Wooi; Harrop, Caroline L.; Bengtsson, Jesse D.; Carvalho, Claudia M. B.; Pehlivan, Davut; Suter, Bernhard; Neul, Jeffrey L.; Liu, Zhandong; Jafar-Nejad, Paymaan; Rigo, Frank; Zoghbi, Huda Y. 分享 收藏
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Targeting the transferrin receptor to transport antisense oligonucleotides across the mammalian blood-brain barrier Barker, Scarlett J.; Thayer, Mai B.; Kim, Chaeyoung; Tatarakis, David; Simon, Matthew J.; Dial, Rebekah; Nilewski, Lizanne; Wells, Robert C.; Zhou, Yinhan; Afetian, Megan; Akkapeddi, Padma; Chappell, Alfred; Chew, Kylie S.; Chow, Johann; Clemens, Allisa; Discenza, Claire B.; Dugas, Jason C.; Dwyer, Chrissa; Earr, Timothy; Ha, Connie; Ho, Yvonne S.; Huynh, David; Lozano, Edwin I.; Jayaraman, Srini; Kwan, Wanda; Mahon, Cathal; Pizzo, Michelle; Robles-Colmenares, Yaneth; Roche, Elysia; Sanders, Laura; Stergioulis, Alexander; Tong, Raymond; Tran, Hai; Zuchero, Joy Yu; Estrada, Anthony A.; Gadkar, Kapil; Koth, Christopher M. M.; Sanchez, Pascal E.; Thorne, Robert G.; Watts, Ryan J.; Sandmann, Thomas; Kane, Lesley A.; Rigo, Frank; Dennis, Mark S.; Lewcock, Joseph W.; Devos, Sarah L. 分享 收藏
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G2C4 targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in human C9orf72 ALS/FTD induced pluripotent stem cell derived neurons Rothstein, Jeffrey D.; Baskerville, Victoria; Rapuri, Sampath; Mehlhop, Emma; Jafar-Nejad, Paymaan; Rigo, Frank; Bennett, Frank; Mizielinska, Sarah; Isaacs, Adrian; Coyne, Alyssa N. 分享 收藏
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Reduction of nemo-like kinase increases lysosome biogenesis and ameliorates TDP-43-related neurodegeneration Tejwani, Leon; Jung, Youngseob; Kokubu, Hiroshi; Sowmithra, Sowmithra; Ni, Luhan; Lee, Changwoo; Sanders, Benjamin; Lee, Paul J.; Xiang, Yangfei; Luttik, Kimberly; Soriano, Armand; Yoon, Jennifer; Park, Junhyun; Ro, Hannah H.; Ju, Hyoungseok; Liao, Clara; Tieze, Sofia Massaro; Rigo, Frank; Jafar-Nejad, Paymaan; Lim, Janghoo 分享 收藏
Genetic Downregulation of the Metabotropic Glutamate Receptor Type 5 Dampens the Reactive and Neurotoxic Phenotype of Adult ALS Astrocytes 代谢型谷氨酸受体5型的遗传下调抑制了成年ALS星形胶质细胞的反应性和神经毒性表型 Torazza, Carola; Provenzano, Francesca; Gallia, Elena; Cerminara, Maria; Balbi, Matilde; Bonifacino, Tiziana; Tessitore, Sara; Ravera, Silvia; Usai, Cesare; Musante, Ilaria; Puliti, Aldamaria; Van Den Bosch, Ludo; Jafar-nejad, Paymaan; Rigo, Frank; Milanese, Marco; Bonanno, Giambattista 分享 收藏
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Differential effects of SARM1 inhibition in traumatic glaucoma and EAE optic neuropathies Liu, Pingting; Chen, Wei; Jiang, Haowen; Huang, Haoliang; Liu, Liping; Fang, Fang; Li, Liang; Feng, Xue; Liu, Dong; Dalal, Roopa; Sun, Yang; Jafar-Nejad, Paymaan; Ling, Karen; Rigo, Frank; Ye, Jiangbin; Hu, Yang 分享 收藏
Antisense oligonucleotides targeting microRNA binding sites in the GRN mRNA increase progranulin translation: potential therapeutic strategy for frontotemporal dementia Jones, Spencer; Aggarwal, Geetika; Benchaar, Yousri; Belanger, Jasmine; Sevigny, Myriam; Smith, Denise; Niehoff, Michael; de Vera, Ian Mitchelle; Petkau, Terri; Leavitt, Blair; Ling, Karen; Jafar-Nejad, Paymaan; Rigo, Frank; Morley, John; Nguyen, Andrew 分享 收藏
ASO silencing of a glycosyltransferase, Poglut1, improves the liver phenotypes in mouse models of Alagille syndrome Niknejad, Nima; Fox, Duncan; Burwinkel, Jennifer L.; Zarrin-Khameh, Neda; Cho, Soomin; Soriano, Armand; Cast, Ashley E.; Lopez, Mario F.; Huppert, Kari A.; Rigo, Frank; Huppert, Stacey S.; Jafar-Nejad, Paymaan; Jafar-Nejad, Hamed 分享 收藏
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