Author Correction: In vivo base editing gene therapy for heterozygous familial hypercholesterolemia: a phase 1 trial.
P. Wan, Si-Yuan Tang, D. Lin et al.
· Nature Medicine · 0 citations
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Oct 2026
In vivo gene editing with YOLT-201 for transthyretin amyloid cardiomyopathy – an open-label, first-in-human clinical trial
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive and life-threatening disease driven by continuous hepatic production of misfolded transthyretin. Existing therapies slow progression but require chronic administration. YOLT-201 is an in vivo CRISPR-Cas9 gene-editing therapy delivered by lipid nanopa...
X. Guo, H. Yan, J.-Y. Liao et al.
· European Heart Journal · 0 citations