Clinical Scorecard: Correction: Phase 1 Trial of In Vivo Base-Editing Gene Therapy for Heterozygous Familial Hypercholesterolemia
At a Glance
Category
Detail
Condition
Clinical topic addressed by the source article: Correction
Key Mechanisms
Correction to reported baseline ApoB and ApoA1 measurements and net background-corrected A-to-G editing values.
Target Population
The correction states that patient eligibility was unaffected.
Care Setting
The correction states that trial procedures and treatment delivery were unaffected.
Key Highlights
Table 1 was corrected after a unit-conversion transcription error placed ApoB and ApoA1 entries against the neighboring non-HDL-C and HDL-C rows, respectively.
Corrected ApoB values for Patients 1–6 are 164, 165, 119, 115, 104 and 171 mg/dL.
Corrected ApoA1 values for Patients 1–6 are 159, 174, 117, 157, 140 and 192 mg/dL.
Extended Data Fig. 2d and its source data were amended to show net background-corrected A-to-G editing values.
The correction states that conclusions, interpretation, results, safety and efficacy assessments, and specified trial methods were unchanged.
Guideline-Based Recommendations
Diagnosis
Management
Monitoring & Follow-up
Risks
Patient & Prescribing Data
Six patients in the reported phase 1 trial; the correction states that patient eligibility was unaffected.
The correction reports no changes to dosing or treatment delivery and provides no new prescribing recommendations.
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