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50 积分 2025-03-14 加入
Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials
1个月前
已完结
CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat β-Thalassemia
1个月前
已完结
AAV-mediated exon skipping therapy for Usher syndrome, type 2A
2个月前
已完结
CRISPR technology: A decade of genome editing is only the beginning
3个月前
已完结
Genome-editing medicinal products: the EMA perspective
3个月前
已完结
Global burden of sickle cell disease in 2021
3个月前
已完结
A practical guide to therapeutic drug monitoring in busulfan: recommendations from the Pharmacist Committee of the European Society for Blood and Marrow Transplantation (EBMT)
4个月前
已完结
Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3
5个月前
已完结
Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3
6个月前
已完结
A human APOC3 missense variant and monoclonal antibody accelerate apoC-III clearance and lower triglyceride-rich lipoprotein levels
6个月前
已完结
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