Rett Syndrome High-Throughput Screening Data for Drug and siRNA Candidates

A screening platform developed in 2026 by Yanhong Yin facilitates treatment development for Rett syndrome. The dataset likely contains results from a high-throughput screening of 1,134 FDA-approved drugs and 336 siRNA genes targeting mitochondrial dysfunction in Rett syndrome astrocytes. It provides candidate drugs and genes identified for reversing mitochondrial membrane potential deficits.

Use Cases

• Identifying candidate drugs for Rett syndrome based on screening results from 1,134 FDA-approved compounds
• Evaluating gene targets for mitochondrial dysfunction based on siRNA knockdown screening of 336 upregulated genes
• Developing novel therapeutic targets for follow-up studies based on the high-throughput screening platform
• Investigating mitochondrial health in neurodevelopmental disorders using JC-10 assay data

Strengths

• Screening results are validated by independent functional assays
• Includes results from a defined screening of 1,134 FDA-approved drugs and 336 siRNA genes
• Platform is based on a JC-10 mitochondrial membrane potential assay serving as a rapid primary readout

Limitations

• Column-level documentation is absent; field semantics must be inferred after download
• Row count is unknown, which may limit suitability assessment
• Data is contained in a PDF file (3.6 MB), which may require extraction for analysis

Provenance

Source

figshare

Collection Method

High-throughput screening based on JC-10 mitochondrial membrane potential assay in astrocytes differentiated from RTT patient-specific induced pluripotent stem cells

Freshness

Last updated 2026-05-08 14:37:44