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

Genetic therapies for neurological diseases

神经系统疾病的基因疗法

Rahim, Ahad A; Kurian, Manju A; Zhou, Haiyan; Ferguson, Ross; Tabrizi, Sarah J; Lignani, Gabriele; Aquilina, Kristian; Waddington, Simon N

MDFIC2 is a PIEZO channel modulator that can alleviate mechanical allodynia associated with neuropathic pain

MDFIC2 是一种压电通道调节器,可以缓解与神经性疼痛相关的机械性痛觉过敏。

Habib, Abdella M; Li, Shengnan; Zhang, Chenjing; Ji, Meijun; Osorio, Nancy; Penalba, Virginie; Torres, Jesus M; Gossage, Samuel J; Rezai, Mehdi A; Geard, Amy F; Rahim, Ahad A; Mahmoud, Ahmed M M; Santana-Varela, Sonia; Zhou, Jun; Zhao, Jing; Wood, John N; Okorokov, Andrei L; Zhou, Xuelong; Cox, James J; Coste, Bertrand

Generation and Treatment of a Novel Severe Model of Visceral Gaucher Disease by Genetic Therapy.

利用基因疗法构建和治疗新型严重内脏戈谢病模型

Geard Amy F, Massaro Giulia, Hughes Michael P, Arbuthnot Patrick, Waddington Simon N, Rahim Ahad A

Engineering an Autonucleolytic Mammalian Suspension Host Cell Line to Reduce DNA Impurity Levels in Serum-Free Lentiviral Process Streams

构建自核酸酶哺乳动物悬浮宿主细胞系以降低无血清慢病毒工艺流程中的DNA杂质水平

Howe, Geoffrey; Wasmuth, Matthew; Emanuelle, Pamela; Massaro, Giulia; Rahim, Ahad A; Ali, Sadfer; Rivera, Milena; Ward, John; Keshavarz-Moore, Eli; Mason, Chris; Nesbeth, Darren N

Fetal gene therapy

胎儿基因治疗

Waddington, Simon N; Peranteau, William H; Rahim, Ahad A; Boyle, Ashley K; Kurian, Manju A; Gissen, Paul; Chan, Jerry K Y; David, Anna L

Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development

溶酶体贮积症的基因治疗:正在进行的研究和临床开发

Massaro, Giulia; Geard, Amy F; Liu, Wenfei; Coombe-Tennant, Oliver; Waddington, Simon N; Baruteau, Julien; Gissen, Paul; Rahim, Ahad A

Correction: Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload

更正:GBA1耗竭导致的细胞生物能量学受损会使神经元对钙超载更加敏感。

Plotegher, Nicoletta; Perocheau, Dany; Ferrazza, Ruggero; Massaro, Giulia; Bhosale, Gauri; Zambon, Federico; Rahim, Ahad A; Guella, Graziano; Waddington, Simon N; Szabadkai, Gyorgy; Duchen, Michael R

Gene therapy for global brain diseases: one small step for mice, one giant leap for humans

基因疗法治疗全球脑部疾病:对小鼠而言是一小步,对人类而言却是一大步。

Rahim, Ahad A; Gissen, Paul

Clinical and Molecular Features of Early Infantile Niemann Pick Type C Disease

早期婴儿尼曼-匹克C型疾病的临床和分子特征

Seker Yilmaz, Berna; Baruteau, Julien; Rahim, Ahad A; Gissen, Paul

Neuroprotective Effects of Diabetes Drugs for the Treatment of Neonatal Hypoxia-Ischemia Encephalopathy

糖尿病药物对新生儿缺氧缺血性脑病的神经保护作用

Poupon-Bejuit, Laura; Rocha-Ferreira, Eridan; Thornton, Claire; Hagberg, Henrik; Rahim, Ahad A