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

Loss of Sarm1 does not suppress motor neuron degeneration in the SOD1G93A mouse model of amyotrophic lateral sclerosis

在肌萎缩侧索硬化症的SOD1G93A小鼠模型中,Sarm1的缺失并不能抑制运动神经元的退化。

Peters, Owen M; Lewis, Elizabeth A; Osterloh, Jeannette M; Weiss, Alexandra; Salameh, Johnny S; Metterville, Jake; Brown, Robert H; Freeman, Marc R

Mutant Profilin1 transgenic mice recapitulate cardinal features of motor neuron disease.

突变型Profilin1转基因小鼠重现了运动神经元疾病的主要特征

Fil Daniel, DeLoach Abigail, Yadav Shilpi, Alkam Duah, MacNicol Melanie, Singh Awantika, Compadre Cesar M, Goellner Joseph J, O'Brien Charles A, Fahmi Tariq, Basnakian Alexei G, Calingasan Noel Y, Klessner Jodi L, Beal Flint M, Peters Owen M, Metterville Jake, Brown Robert H Jr, Ling Karen K Y, Rigo Frank, Ozdinler P Hande, Kiaei Mahmoud