articleNature MedicineMar 1, 2026Closed access

In vivo base editing gene therapy for heterozygous familial hypercholesterolemia: a phase 1 trial

Shanghai Jiao Tong University · Renji Hospital · +6 more institutions

PubMed
Indexed incrossrefpubmed

Abstract

No abstract available for this paper.

Citation impact

5
total citations
FWCI
76.09
Percentile
100%
References
39
Too recent for citation history.

Authors

16

Topics & keywords

Keywords
  • Familial hypercholesterolemia
  • PCSK9
  • In vivo
  • Genetic enhancement
  • Adverse effect
  • Clinical trial
  • Cholesterol
  • Genome editing
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