SOD1-Derlin-1 inhibitor-1;8404

Lentiviral and Adeno-Associated Vector-Based Therapy for Motor Neuron Disease Through RNAi

virus have been used to knockdown levels of mutated superoxide dismutase 1 (SOD1) in the G93A SOD1 mouse model of familial amyotrophic lateral sclerosis (fALS) to result in beneficial therapeutic outcomes. This chapter describes the design, production

Analysis of CFTR Folding and Degradation in Transiently Transfected Cells

. The folding status of F508del-CFTR is monitored by at least two ER quality control checkpoints. The ER-associated Derlin-1/RMA1 E3 complex appears to recognize folding defects in CFTR that involve misassembly of NBD1 into a complex with the R-domain

RNAi治疗肌萎缩侧索症

发表在4月出版的《自然—医学》上的两份报告显示，核糖核酸(RNA)干扰也许是治疗肌萎缩侧索硬化症(ALS)的一种有效方法。 ALS也称为格里克症，表现为脊柱神经的死亡所导致的肌肉消瘦和运动问题。遗传性ALS的发病原因是一种名为SOD1的蛋白质的基因发生了变异，经过基因工程携带上一个这种人类基因的小鼠出现了类似于ALS患者的症状。Patrick Aebischer和Scott Ralph领导的两个小组独立地显示，发送能抑止SOD1合成的小分子干扰RNAs可推迟这种小鼠