Lv41
550 积分 2025-03-16 加入
Inhibition of Hedgehog signaling does not mitigate polycystic kidney disease severity in a Pkd1 mutant mouse model
2小时前
待确认
Applications of gene therapy to kidney disease
26天前
已完结
Infarct of the prostate
1个月前
已关闭
AAV9-Mediated Gene Therapy for Infantile-Onset Pompe’s Disease
1个月前
已完结
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial
1个月前
已完结
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
1个月前
已关闭
AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial
1个月前
已完结
Base editing of trinucleotide repeats that cause Huntington’s disease and Friedreich’s ataxia reduces somatic repeat expansions in patient cells and in mice
1个月前
已完结
Lentiviral gene therapy with reduced-intensity conditioning for sickle cell disease: a phase 1/2 trial
1个月前
已完结
Treating genetic blood disorders in the era of CRISPR-mediated genome editing
1个月前
已关闭
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