Lv5
815 积分 2022-02-16 加入
Long-term reversal of Duchenne muscular dystrophy via circular arRNA-guided exon skipping in monkeys and humans
4天前
已完结
AAV-mediated CBLN1 replacement rescues hereditary ataxia caused by biallelic CBLN1 variants
7天前
已完结
CDKL5 deficiency disorder: molecular insights and mechanisms of pathogenicity to fast-track therapeutic development
15天前
已关闭
Extracellular vesicles and mitochondria in central nervous system diseases
26天前
已完结
Recent advancements in improving cross-species applicability of bioengineered AAV capsids
2个月前
已完结
In Vivo Genome Editing Approach to Disrupt Hydroxyacid Oxidase 1 for the Treatment of Primary Hyperoxaluria Type 1
2个月前
已完结
Adeno-Associated Virus Gene Therapy Translation: Lessons from Early Regulatory Meetings
2个月前
已完结
Dual-target gene therapy in Parkinson’s disease: a multicenter phase 1 trial
3个月前
已完结
Long-term reversal of Duchenne muscular dystrophy via circular arRNA-guided exon skipping in monkeys and humans
3个月前
已完结
At the forefront of gene-based therapies in dravet syndrome
3个月前
已完结
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