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

Nature Medicine

Overview

A Nature Medicine correction amended baseline apolipoprotein B (ApoB) and apolipoprotein A1 (ApoA1) values in Table 1 and corrected the presentation of A-to-G editing frequencies in Extended Data Fig. 2d. The correction states that the article’s conclusions, interpretation, results, safety and efficacy assessments, and trial methods were unchanged.

Background

The correction addresses two reporting errors in the phase 1 trial article. During conversion from g/L to mg/dL, a transcription mistake placed the baseline ApoB and ApoA1 entries against the neighboring non-HDL-C and HDL-C rows, respectively. Extended Data Fig. 2d initially showed A-to-G editing frequencies before subtraction of matched untreated-control background, although its legend described background-corrected measurements.

Data Highlights

Measure	Patients 1–6, corrected values	Correction
ApoB (mg/dL)	164, 165, 119, 115, 104, 171	Corrected baseline entries in Table 1
ApoA1 (mg/dL)	159, 174, 117, 157, 140, 192	Corrected baseline entries in Table 1
A-to-G editing frequencies	Net background-corrected values	Extended Data Fig. 2d and associated source data revised

Key Findings

Table 1 was amended to correct baseline ApoB and ApoA1 measurements for Patients 1–6.
The correction attributes the row-placement errors to a transcription mistake during conversion from g/L to mg/dL.
Extended Data Fig. 2d was amended to show A-to-G editing frequencies after subtraction of matched untreated-control background, as described in the figure legend.
The associated source data for Extended Data Fig. 2d were revised in parallel.
The correction states that the article’s conclusions, interpretation, results, and safety and efficacy assessments were unchanged.
The correction states that the underlying sequencing data, off-target evaluation, patient eligibility, trial procedures, dosing, treatment delivery, and statistical analyses were unaffected.

Clinical Implications

The correction revises the baseline ApoB and ApoA1 entries in Table 1 and updates Extended Data Fig. 2d and its associated source data to show background-corrected A-to-G editing frequencies. The correction states that the article’s conclusions, interpretation, results, and safety and efficacy assessments were unchanged.

Conclusion

The correction amended baseline ApoB and ApoA1 measurements in Table 1 and corrected the presentation of A-to-G editing frequencies in Extended Data Fig. 2d. It states that the article’s conclusions, interpretation, results, and safety and efficacy assessments were unchanged.