Lv1
30 积分 2023-04-02 加入
Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems
8天前
已完结
Long-term reversal of Duchenne muscular dystrophy via circular arRNA-guided exon skipping in monkeys and humans
1个月前
已完结
Syndromes of Thrombotic Microangiopathy
2个月前
已完结
Low-dose AAV9-SMN1 with CNS-selective expression delivers efficacy and favorable safety in spinal muscular atrophy
2个月前
已完结
Neuroepithelial Tumor with AAV Integration after Intracisternal Magna Vector Delivery
2个月前
已完结
Neuroepithelial Tumor with AAV Integration after Intracisternal Magna Vector Delivery
2个月前
已完结
Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial
3个月前
已完结
Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial
3个月前
已完结
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease
4个月前
已完结
Transduction of Quiescent Human Hematopoietic Stem and Progenitor Cells Using Lentiviral Vectors and Virus‐Like Particles
4个月前
已完结
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