日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Acute neuronal inhibition response caused by phosphorothioate antisense oligonucleotides following local delivery to the central nervous system

局部递送硫代磷酸酯反义寡核苷酸至中枢神经系统后引起的急性神经元抑制反应

O'Rourke, Jacqueline G; Bachmann, Gemma; Mazur, Curt; Zhou, Keming; Platoshyn, Oleksandr; Bravo Hernandez, Mariana; Klein, Stephanie K; Nguyen, Jonathon; Burel, Sebastien; Hoffmaster, Christine; Zanardi, Thomas; Jafar-Nejad, Paymaan; Marsala, Martin; Henry, Scott P; Swayze, Eric E; Powers, Berit; Kordasiewicz, Holly B

Transient acute neuronal activation response caused by high concentrations of oligonucleotides in the cerebral spinal fluid

脑脊液中高浓度寡核苷酸引起的短暂急性神经元激活反应

Bravo-Hernandez, Mariana; Mazur, Curt; Chen, Hao; Fradkin, Linda; Searcy, Justin; Burel, Sebastien; Kelly, Mackenzie; Bruening, Dona; O'Rourke, Jacqueline G; Cai, Yuhang; Nguyen, Jonathon; Berman-Booty, Lisa; Cummins, Lendell; Gaus, Hans; Powers, Berit; Zhao, Hien T; Jafar-Nejad, Paymaan; Henry, Scott; Swayze, Eric E; Kordasiewicz, Holly B

Discovery of long-acting APOC3 siRNA for treating patients with hypertriglyceridemia

发现长效APOC3 siRNA用于治疗高甘油三酯血症患者

Prakash, Thazha P; Mullick, Adam E; Riney, Stan; Yu, Jinghua; Nikan, Mehran; Quirk, Clare; Crutchfield, Amanda; Damle, Sagar S; Klein, Stephanie; Swayze, Eric E

Single alkyl phosphonate modification of the siRNA backbone in the seed region enhances specificity and therapeutic profile

在siRNA种子区对siRNA骨架进行单烷基膦酸酯修饰可增强其特异性和治疗效果。

Nikan, Mehran; Li, Qingfeng; Tanowitz, Michael; Li, Hongda; Damle, Sagar; Annoual, Marie; Galindo-Murillo, Rodrigo; Low, Audrey; Klein, Stephanie; Quirk, Clare; Vasquez, Guillermo; Wan, W Brad; Watt, Andrew T; Migawa, Michael T; Swayze, Eric E; Prakash, Thazha P

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结合的双环肽偶联可增强ASO和siRNA在骨骼肌和心肌中的效力。

Østergaard, 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; Chen, Liuhong; Skynner, Michael J; Swayze, Eric E

Author Correction: Tau-targeting antisense oligonucleotide MAPT(Rx) in mild Alzheimer's disease: a phase 1b, randomized, placebo-controlled trial

作者更正:靶向Tau蛋白的反义寡核苷酸MAPT(Rx)治疗轻度阿尔茨海默病:一项1b期随机安慰剂对照试验

Mummery, Catherine J; Börjesson-Hanson, Anne; Blackburn, Daniel J; Vijverberg, Everard G B; De Deyn, Peter Paul; Ducharme, Simon; Jonsson, Michael; Schneider, Anja; Rinne, Juha O; Ludolph, Albert C; Bodenschatz, Ralf; Kordasiewicz, Holly; Swayze, Eric E; Fitzsimmons, Bethany; Mignon, Laurence; Moore, Katrina M; Yun, Chris; Baumann, Tiffany; Li, Dan; Norris, Daniel A; Crean, Rebecca; Graham, Danielle L; Huang, Ellen; Ratti, Elena; Bennett, C Frank; Junge, Candice; Lane, Roger M

Tau-targeting antisense oligonucleotide MAPT(Rx) in mild Alzheimer's disease: a phase 1b, randomized, placebo-controlled trial

靶向Tau蛋白的反义寡核苷酸MAPT(Rx)治疗轻度阿尔茨海默病:一项1b期随机、安慰剂对照试验

Mummery, Catherine J; Börjesson-Hanson, Anne; Blackburn, Daniel J; Vijverberg, Everard G B; De Deyn, Peter Paul; Ducharme, Simon; Jonsson, Michael; Schneider, Anja; Rinne, Juha O; Ludolph, Albert C; Bodenschatz, Ralf; Kordasiewicz, Holly; Swayze, Eric E; Fitzsimmons, Bethany; Mignon, Laurence; Moore, Katrina M; Yun, Chris; Baumann, Tiffany; Li, Dan; Norris, Daniel A; Crean, Rebecca; Graham, Danielle L; Huang, Ellen; Ratti, Elena; Bennett, C Frank; Junge, Candice; Lane, Roger M

Mechanisms of palmitic acid-conjugated antisense oligonucleotide distribution in mice

棕榈酸偶联反义寡核苷酸在小鼠体内的分布机制

Chappell, Alfred E; Gaus, Hans J; Berdeja, Andres; Gupta, Ruchi; Jo, Minji; Prakash, Thazha P; Oestergaard, Michael; Swayze, Eric E; Seth, Punit P

Likelihood of Nonspecific Activity of Gapmer Antisense Oligonucleotides Is Associated with Relative Hybridization Free Energy

Gapmer反义寡核苷酸的非特异性活性可能性与相对杂交自由能相关。

Watt, Andrew T; Swayze, Grant; Swayze, Eric E; Freier, Susan M

Site-specific replacement of phosphorothioate with alkyl phosphonate linkages enhances the therapeutic profile of gapmer ASOs by modulating interactions with cellular proteins

通过位点特异性地用烷基膦酸酯键取代硫代磷酸酯键,可以调节间隙型反义寡核苷酸(ASO)与细胞蛋白的相互作用,从而增强其治疗效果。

Migawa, Michael T; Shen, Wen; Wan, W Brad; Vasquez, Guillermo; Oestergaard, Michael E; Low, Audrey; De Hoyos, Cheryl L; Gupta, Ruchi; Murray, Susan; Tanowitz, Michael; Bell, Melanie; Nichols, Joshua G; Gaus, Hans; Liang, Xue-Hai; Swayze, Eric E; Crooke, Stanley T; Seth, Punit P