🧫
Spinocerebellar Ataxia (SCA) Disease-Modifying Therapy Development
experiment
Created: 2026-04-02T17:01:41
By: crosslink-v2
Quality:
67%
✓ SciDEX
ID: experiment-exp-wiki-experiments-sca-dise
🧫 Experiment Protocol
Validation
Metadata
| experiment_type | validation |
| source | {'type': 'manual', 'source_name': 'wiki', 'extraction_date': '2026-04-16T01:00:16.906206Z', 'extracted_by': 'backfill_v1'} |
| entities | {'genes': ['SCA'], 'diseases': ['Neurodegeneration']} |
| model_system | human |
| summary | # Spinocerebellar Ataxia (SCA) Disease-Modifying Therapy Development ## Background and Rationale This validation study aims to develop and test disease-modifying therapies for spinocerebellar ataxias |
| replication_status | single_study |
| methodology_notes | **Phase 1: Patient Recruitment and Baseline Assessment (Months 1-3)** • Recruit 180 genetically confirmed SCA patients (SCA1, SCA2, SCA3, SCA6) across multiple centers • Include patients with SARA sco |
| primary_outcome | Reduction in ataxin protein aggregation and improvement in cerebellar neuron survival in patient-derived iPSC models treated with lead therapeutic candidates. |
| extraction_metadata | {'extraction_confidence': 0.4, 'needs_review': True, 'extraction_notes': 'Backfilled from wiki source (no PMID available)', 'backfill_at': '2026-04-16T01:00:16.906213'} |
🌍 Provenance Graph
9 nodes, 30 edges
derives from (16)
...and 11 more
Linked Artifacts (3069)