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2 积分 2023-09-18 加入
Dual-target gene therapy in Parkinson’s disease: a multicenter phase 1 trial
26天前
已完结
Long-term reversal of Duchenne muscular dystrophy via circular arRNA-guided exon skipping in monkeys and humans
27天前
已完结
Prime editing of a pathogenic Scn1a allele ameliorates seizure phenotypes in a GEFS + mouse model
1个月前
已完结
In vivo adenine base editing ameliorates Dravet syndrome phenotypes in a mouse model
1个月前
已完结
Mechanism of STMN2 cryptic splice-polyadenylation and its correction for TDP-43 proteinopathies
1个月前
已完结
Identifying genome-wide off-target sites of CRISPR RNA–guided nucleases and deaminases with Digenome-seq
1个月前
已完结
Short RNA chaperones promote aggregation-resistant TDP-43 conformers to mitigate neurodegeneration
1个月前
已完结
Translation-dependent degradation of cas12 mRNA triggered by an anti-CRISPR
2个月前
已完结
Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells
2个月前
已完结
Multicentre gene therapy for OTOF-related deafness followed up to 2.5 years
2个月前
已完结
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