# Correction Issued for Phase 1 Gene Therapy Trial Data Researchers rectify transcription errors in clinical trial data for familial hypercholesterolemia gene therapy. - **Source:** [Nature Medicine](https://www.nature.com/nm) - **Published:** 2026-11-03 - **Topic:** [health](https://brifing.si/c/health) - **Original article:** - **On Brifing:** https://brifing.si/s/c21cb16e-a3a1-4771-8e71-f0958254cab0 ## Correction 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. ## Specific 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. ## Figure 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. ## Scope 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. ## Institutional 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. ## Base 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. ## Clinical 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. ## Implications 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. ## Sources covering this story - https://www.nature.com/articles/s41591-026-04786-9 — --- _Summary written by Brifing from the original reporting. Copyright in the original article belongs to its publisher; read the full piece at the link above._