Human HAO1: Target Enabling Package for Drug Discovery

Sabrina MacKinnon at the University of Oxford provides a Target Enabling Package (TEP) for developing small molecule inhibitors against the enzyme Hydroxyacid Oxidase 1 (HAO1). The dataset includes recombinant protein purification protocols, enzyme structures in different states, in vitro assay data for ligand binding and activity, and initial chemical matter from crystallography-based fragment screening. This resource supports a substrate reduction therapy approach for treating Primary Hyperoxaluria type 1, an inherited metabolic disorder.

Use Cases

• Identifying novel HAO1 inhibitors based on fragment screening data.
• Validating computational docking or binding affinity predictions using provided SPR and DSF assay results.
• Studying enzyme-ligand interactions using the provided HAO1 protein structures.
• Developing substrate reduction therapies for Primary Hyperoxaluria type 1 based on the described metabolic pathway target.

Strengths

• Includes multiple experimental data modalities: protein structures, biochemical assay data (SPR, DSF, Amplex Red), and fragment screening results.
• Provides a clear therapeutic context and biological rationale targeting the HAO1 enzyme for a specific genetic disorder.
• Originates from a recognized academic institution (University of Oxford).

Limitations

• Key metadata such as row count, specific column names, and file formats are not provided across any source.
• The exact date of the last update is unknown, limiting assessment of data freshness.
• The dataset's scale and the number of unique chemical fragments screened are unspecified.

Provenance

Source

Sabrina MacKinnon, University of Oxford

Collection Method

Experimental data collection including recombinant protein purification, X-ray crystallography, and in vitro biochemical assays (DSF, SPR, Amplex Red).