CRISPR-Cas9 Gene Editing Efficacy Data for Familial Hypercholesterolemia Mouse Model

41.3% frameshift indels in liver tissue were achieved via CRISPR-Cas9 targeting of mutant PCSK9, leading to a 78.5% reduction in serum PCSK9 and a 67.2% reduction in LDL cholesterol sustained for 24 weeks. The dataset, published by Ge Ali on figshare under CC-BY-4.0, contains results from an in vivo study using an AAV9 vector to deliver SaCas9. Whole-genome sequencing of predicted off-target sites revealed no detectable mutations, and no liver toxicity was observed.

Use Cases

• Validate gene editing efficiency based on amplicon deep sequencing results showing indel rates
• Model therapeutic lipid-lowering effects based on serum PCSK9 and LDL cholesterol reduction data
• Assess off-target risk for CRISPR-Cas9 therapies based on whole-genome sequencing results
• Study the durability of gene correction based on 24-week persistence data

Strengths

• Specific, quantified outcomes: 41.3% frameshift indels, 78.5% PCSK9 reduction, 67.2% LDL cholesterol reduction
• Long-term efficacy data spanning 24 weeks
• Safety data includes whole-genome off-target analysis and liver toxicity assessment

Limitations

• Row count is unknown, which may limit suitability assessment
• Column-level documentation is absent; field semantics must be inferred after download
• The dataset is very small at 13.3 KB, indicating limited scope

Provenance

Source

figshare

Collection Method

Experimental data from an in vivo mouse model study using AAV9-delivered CRISPR-Cas9.

Freshness

Last updated 2026-05-30 13:35:18; freshness should be verified