Hemophilia A Mouse Model: Long-Term Outcomes of Minicircle FVIII Gene Therapy

A mouse model study evaluating non-viral gene therapy for hemophilia A. Yung-Tsung Kao authored this dataset, which contains outcomes from minicircle DNA delivery of Factor VIII over 26 weeks. The data was last updated on April 9, 2026.

Use Cases

• Evaluate coagulation correction efficacy based on aPTT and FVIII activity measurements
• Assess transgene persistence based on DNA and RNA detection in hepatocytes
• Compare durability of gene therapy versus conventional infusion based on the 26-week monitoring period

Strengths

• Data covers a 26-week longitudinal monitoring period
• Includes specific coagulation outcomes: aPTT, FVIII activity, and tail-clip assay results
• License is CC-BY-4.0, permitting open reuse

Limitations

• Row count is unknown, which may limit suitability assessment
• Column-level documentation is absent; field semantics must be inferred after download
• Dataset size is 305.3 KB, indicating a limited scope

Provenance

Source

figshare

Collection Method

Hydrodynamic tail vein injection of minicircle DNA constructs into FVIII-KO mice

Time Range

26-week monitoring period

Freshness

Last updated 2026-04-09 05:14:52