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

Complement activation in a phase Ib study of fordadistrogene movaparvovec for Duchenne muscular dystrophy

在针对杜氏肌营养不良症的 fordadistrogene movaparvovec 的 Ib 期研究中,补体激活

Byrne, Barry J; Butterfield, Russell J; Shieh, Perry B; Smith, Edward C; Licht, Christoph; Binks, Michael; Casinghino, Sandra; Delnomdedieu, Marielle; Ravindra, Kodihalli C; McDonnell, Tara; Ryan, Kelly; Schulz, Martin; Shen, Qi; Shi, Heliang; Sirivelu, Madhu P; Vaidya, Vishal S; Whiteley, Laurence; Levy, Daniel I

Single cell and TCR analysis of immune cells from AAV gene therapy-dosed Duchenne muscular dystrophy patients

对接受AAV基因治疗的杜氏肌营养不良症患者的免疫细胞进行单细胞和TCR分析

Emami, Michael R; Brimble, Mark A; Espinoza, Alejandro; Owens, Jane; Whiteley, Laurence O; Casinghino, Sandra; Lanz, Thomas A; Farahat, Philip K; Pellegrini, Matteo; Young, Courtney S; Thomas, Paul G; McNally, Elizabeth M; Villalta, S Armando; Schattgen, Stefan A; Spencer, Melissa J

Complement Activation by Adeno-Associated Virus-Neutralizing Antibody Complexes

腺相关病毒-中和抗体复合物激活补体

West, Cara; Federspiel, Joel D; Rogers, Kara; Khatri, Arpana; Rao-Dayton, Sheila; Ocana, Mireia Fernandez; Lim, Sean; D'Antona, Aaron Michael; Casinghino, Sandra; Somanathan, Suryanarayan

Biodistribution and Tolerability of AAV-PHP.B-CBh- SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles

AAV-PHP.B-CBh-SMN1 在 Wistar Han 大鼠和食蟹猴体内的生物分布和耐受性显示出不同的毒理学特征

Xavier Palazzi, Ingrid D Pardo, Madhu P Sirivelu, Leah Newman, Steven W Kumpf, Jessie Qian, Tania Franks, Sarah Lopes, June Liu, Laura Monarski, Sandra Casinghino, Casey Ritenour, Hayley Ritenour, Christopher Dubois, Jennifer Olson, John Graves, Kristin E Alexander, Timothy Coskran, Thomas A Lanz, J