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

RNA editing for the treatment of alpha-1 antitrypsin deficiency.

RNA 编辑用于治疗 α-1 抗胰蛋白酶缺乏症。

Monian Prashant, Shivalila Chikdu, Lu Genliang, Bowman Keith, Briem Stearne, Bylsma Marissa, Byrne Michael, Cannon Megan, Chatterjee Arindom, Dallaire Morgan, Desai Jigar, Dufresne Jason, Faraone Alyse, Favaloro Frank, Friend Stephen, Ghosh Anamitra, Godfrey Jack, Hernandez Nidia, Huth Olivia, Iwamoto Naoki, Kawamoto Tomomi, Lamattina Anthony, Lemaitre Muriel, Lindsey Amber, Liu Fangjun, Looby Richard, Luu Khoa, McGlynn Andrew, Metterville Jake, Paik Ik-Hyeon, Pan Qianli, Patil Saurabh, Pu Tom, Purcell-Estabrook Erin, Ranade Ashwini, Rheinhardt Jeanette, Rossi Jeff, Shimizu Mamoru, Singh Kuldeep, Standley Stephany, Thomas Carina, Tripathi Snehlata, Yang Hailin, Yordanoff Ryan, Yin Yuan, Yu Hui, Narayanan PadmaKumar, Kandasamy Pachamuthu, Giangrande Paloma H, Vargeese Chandra

Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT

对立体纯反义寡核苷酸进行临床前评估,以选择性降低突变型HTT的表达水平。

Iwamoto, Naoki; Liu, Yuanjing; Frank-Kamenetsky, Maria; Maguire, Abbie; Tseng, Wei Chou; Taborn, Kristin; Kothari, Nayantara; Akhtar, Ali; Bowman, Keith; Shelke, Juili Dilip; Lamattina, Anthony; Hu, Xiao Shelley; Jang, Hyun Gyung; Kandasamy, Pachamuthu; Liu, Fangjun; Longo, Ken; Looby, Richard; Meena; Metterville, Jake; Pan, Qianli; Purcell-Estabrook, Erin; Shimizu, Mamoru; Prakasha, Priyanka Shiva; Standley, Stephany; Upadhyay, Hansini; Yang, Hailin; Yin, Yuan; Zhao, Anderson; Francis, Christopher; Byrne, Mike; Dale, Elena; Verdine, Gregory L; Vargeese, Chandra

High-temperature oxidation kinetics of nanostructured thermoelectric skutterudite CoSb(3) under different environments

不同环境下纳米结构热电方钴矿CoSb(3)的高温氧化动力学

Anusree, T C; Vargeese, Anuj A

Preclinical evaluation of WVE-004, aninvestigational stereopure oligonucleotide forthe treatment of C9orf72-associated ALS or FTD

WVE-004(一种用于治疗 C9orf72 相关 ALS 或 FTD 的试验性立体纯寡核苷酸)的临床前评估

Yuanjing Liu, Amy Andreucci, Naoki Iwamoto, Yuan Yin, Hailin Yang, Fangjun Liu, Alexey Bulychev, Xiao Shelley Hu, Xuena Lin, Sarah Lamore, Saurabh Patil, Susovan Mohapatra, Erin Purcell-Estabrook, Kristin Taborn, Elena Dale, Chandra Vargeese

Quantitative Measurement of Cytosolic and Nuclear Penetration of Oligonucleotide Therapeutics

寡核苷酸疗法胞质和核渗透的定量测定

Deprey, Kirsten; Batistatou, Nefeli; Debets, Marjoke F; Godfrey, Jack; VanderWall, Kirstin B; Miles, Rebecca R; Shehaj, Livia; Guo, Jiaxing; Andreucci, Amy; Kandasamy, Pachamuthu; Lu, Genliang; Shimizu, Mamoru; Vargeese, Chandra; Kritzer, Joshua A

Stereochemistry Enhances Potency, Efficacy, and Durability of Malat1 Antisense Oligonucleotides In Vitro and In Vivo in Multiple Species

立体化学增强了Malat1反义寡核苷酸在体外和体内多种物种中的效力、功效和持久性

Byrne, Michael; Vathipadiekal, Vinod; Apponi, Luciano; Iwamoto, Naoki; Kandasamy, Pachamuthu; Longo, Kenneth; Liu, Fangjun; Looby, Richard; Norwood, Lauren; Shah, Anee; Shelke, Juili Dilip; Shivalila, Chikdu; Yang, Hailin; Yin, Yuan; Guo, Lankai; Bowman, Keith; Vargeese, Chandra

Investigational Assay for Haplotype Phasing of the Huntingtin Gene

亨廷顿基因单倍型定相试验

Nenad Svrzikapa, Kenneth A Longo, Nripesh Prasad, Ramakrishna Boyanapalli, Jeffrey M Brown, Daniel Dorset, Scott Yourstone, Jason Powers, Shawn E Levy, Aaron J Morris, Chandra Vargeese, Jaya Goyal

Dendritic Polynitrato Energetic Motifs: Development and Exploration of Physicochemical Behavior through Theoretical and Experimental Approach

树枝状聚硝酸盐高能结构:通过理论和实验方法研究其物理化学行为

Gaur, Pankaj; Dev, Sagarika; Kumar, Sunil; Kumar, Mahesh; Vargeese, Anuj A; Soni, Pramod; Siril, Prem Felix; Ghosh, Subrata

Efficient activation of nucleoside phosphoramidites with 4,5-dicyanoimidazole during oligonucleotide synthesis

在寡核苷酸合成过程中,利用4,5-二氰基咪唑高效活化核苷亚磷酰胺

Vargeese, C; Carter, J; Yegge, J; Krivjansky, S; Settle, A; Kropp, E; Peterson, K; Pieken, W