Correction Issued for Phase 1 Gene Therapy Trial Data
Researchers rectify transcription errors in clinical trial data for familial hypercholesterolemia gene therapy.
1Correction of Clinical Data
The original article published on March 3, 2026, regarding a phase 1 trial for heterozygous familial hypercholesterolemia, has undergone a formal correction. Researchers identified transcription errors in Table 1 involving baseline apolipoprotein B and apolipoprotein A1 values. These inaccuracies stemmed from unit conversion errors between grams per liter and milligrams per deciliter.
2Specific Data Adjustments
The corrected apolipoprotein B values for the six patients are now listed as 164, 165, 119, 115, 104, and 171 mg/dL. Similarly, the apolipoprotein A1 values were updated to 159, 174, 117, 157, 140, and 192 mg/dL. These adjustments ensure the baseline clinical data accurately reflects the patients' initial physiological profiles during the study.
3Figure Visualization Update
Extended Data Fig. 2d required revision to accurately represent A-to-G editing frequencies. The initial version displayed raw values before subtracting the matched untreated-control background. The updated figure now presents the background-corrected net editing values, aligning the visual data with the descriptions provided in the figure legend and the corresponding source data files.
4Scope of the Correction
The authors confirmed that these specific corrections do not impact the study's fundamental sequencing data. Furthermore, the off-target assessment conclusions remain unchanged. The adjustments do not affect patient eligibility criteria, trial conduct, dose assignments, treatment administration protocols, or the statistical analyses performed. The overall safety and efficacy results remain consistent with the original findings.
5Institutional Research Context
The study was conducted by a multi-institutional team, including researchers from the Department of Liver Surgery at Ren Ji Hospital and the Shanghai Key Laboratory of Precision Gene Editing. Collaborators also included experts from East China Normal University and Hannover Medical School, reflecting a broad international and interdisciplinary effort in gene therapy.
6Base Editing Mechanism
The trial investigates in vivo base editing, a precise gene-editing technology that allows for the direct conversion of one DNA base into another without creating double-strand breaks. This approach is particularly relevant for treating genetic conditions like familial hypercholesterolemia, which is characterized by dangerously high levels of low-density lipoprotein cholesterol in blood.
7Clinical Trial Integrity
Maintaining the accuracy of baseline data is critical in phase 1 trials, which primarily focus on safety and dosage. By promptly addressing transcription errors, the researchers uphold the transparency required for clinical research. Such corrections are standard practice in scientific publishing to ensure that peer-reviewed literature remains a reliable source for future medical developments.
8Implications for Future Research
While these corrections address technical reporting errors, they reinforce the importance of rigorous data management in complex gene therapy trials. As the field of base editing continues to evolve, the clarity of baseline measurements and background-corrected data remains essential for researchers to evaluate the potential long-term therapeutic impact of these novel genetic interventions.
Original article: Nature Medicine — Correction Issued for Phase 1 Gene Therapy Trial Data