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

Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA

慢病毒载体介导的体外干细胞基因治疗治疗粘多糖贮积症IIIA的临床前安全性和有效性

Stuart M Ellison, Aiyin Liao, Shaun Wood, Jessica Taylor, Amir Saam Youshani, Sam Rowlston, Helen Parker, Myriam Armant, Alessandra Biffi, Lucas Chan, Farzin Farzaneh, Rob Wynn, Simon A Jones, Paul Heal, H Bobby Gaspar, Brian W Bigger

DNAJC21 Mutations Link a Cancer-Prone Bone Marrow Failure Syndrome to Corruption in 60S Ribosome Subunit Maturation

DNAJC21 突变将癌症易发性骨髓衰竭综合征与 60S 核糖体亚基成熟过程中的破坏联系起来

Hemanth Tummala, Amanda J Walne, Mike Williams, Nicholas Bockett, Laura Collopy, Shirleny Cardoso, Alicia Ellison, Rob Wynn, Thierry Leblanc, Jude Fitzgibbon, David P Kelsell, David A van Heel, Elspeth Payne, Vincent Plagnol, Inderjeet Dokal, Tom Vulliamy