Toward a Small Molecule Therapy for Angelman Syndrome: Structure–Activity Relationship Stu

Structure–activity relationship data for 3-aminopyrazole phenylacetamide (APPA) compounds tested as unsilencers of paternal Ube3a. The dataset, authored by Anna Welton-Arndt and last updated on 2026-05 08, likely contains results from assays in mouse primary neurons measuring unsilencing behavior and cellular toxicity. The 5.7 KB CSV file originates from research published on figshare.

Use Cases

• Modeling compound potency based on reported structure-activity relationships.
• Analyzing cytotoxicity profiles of APPA scaffold derivatives.
• Identifying promising lead compounds for further preclinical development.
• Studying transcriptional disruption patterns of Ube3a-ATS.

Strengths

• Data is associated with a specific, peer-reviewed research project on a leading therapeutic strategy.
• The dataset is small (5.7 KB), facilitating quick inspection and download.
• It is licensed under CC-BY-NC-4.0, permitting non-commercial reuse with attribution.

Limitations

• Row count is unknown, which may limit suitability assessment.
• Column-level documentation is absent; field semantics must be inferred after download.
• The dataset's scope is limited to a specific chemical scaffold (APPA) and a single reporter assay.

Provenance

Source

figshare

Collection Method

Likely contains experimental results from assays in mouse primary neurons harboring a reporter of paternal Ube3a expression.

Time Range

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Freshness

Last updated 2026-05-08 20:04:45; freshness should be verified.

Geography

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