Author Correction: In vivo base editing gene therapy for heterozygous familial hypercholesterolemia: a phase 1 trial - Summary - MDSpire
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Correction: Phase 1 Trial of In Vivo Base-Editing Gene Therapy for Heterozygous Familial Hypercholesterolemia

  • By

  • Ping Wan

  • Siyuan Tang

  • Dongni Lin

  • Yuming Lu

  • Mei Long

  • Ling Xiao

  • Yanhong Jiang

  • Jiaoyang Liao

  • Xiaoying Ma

  • Ying Liu

  • Wensu Yu

  • Michael Ott

  • Zi Jun Wang

  • Yuxuan Wu

  • Taihua Yang

  • Qiang Xia

  • November 3, 2026

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Objective:

The article examines the objective, findings, and clinical context described in Correction: Phase 1 Trial of In Vivo Base-Editing Gene Therapy for Heterozygous Familial Hypercholesterolemia.

Approach:
  • Table 1: Corrected baseline ApoB and ApoA1 values for Patients 1–6 after a unit-conversion transcription error had placed entries against neighboring lipid rows.
  • Extended Data Fig. 2d: Revised the panel and source data to show net background-corrected A-to-G editing frequencies.
Key Findings:
  • Corrected ApoB values (mg/dL), Patients 1–6: 164, 165, 119, 115, 104, and 171.
  • Corrected ApoA1 values (mg/dL), Patients 1–6: 159, 174, 117, 157, 140, and 192.
  • The original figure showed treated-sample A-to-G frequencies before subtraction of matched untreated-control background, although its legend described background-corrected values.
Interpretation:

The correction states that the article’s conclusions, interpretation, results, and safety and efficacy assessments are unchanged.

Conclusion:

Underlying sequencing data, off-target evaluation, eligibility, trial procedures, dosing, treatment delivery, and statistical analyses were unaffected.

Sources:

Original Source(s)

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