🧫
Progranulin Replacement Therapy for FTD — Vector Development and Validation
experiment
Created: 2026-04-02T17:01:41
By: crosslink-v2
Quality:
67%
✓ SciDEX
ID: experiment-exp-wiki-experiments-progranu
🧫 Experiment Protocol
Clinical
Metadata
| experiment_type | clinical |
| source | {'type': 'manual', 'source_name': 'wiki', 'extraction_date': '2026-04-16T01:00:16.900984Z', 'extracted_by': 'backfill_v1'} |
| entities | {'genes': ['FTD'], 'diseases': ['ALS']} |
| model_system | human |
| summary | # Progranulin Replacement Therapy for FTD — Vector Development and Validation ## Background and Rationale This clinical trial focuses on developing and validating progranulin replacement therapy for f |
| replication_status | conflicting |
| methodology_notes | Phase 1: Participant recruitment and screening (months 1-6) of 60 presymptomatic GRN mutation carriers and 40 early symptomatic patients through established FTD research networks. Inclusion criteria: |
| primary_outcome | Dose-dependent increase in CSF progranulin levels from baseline to 6 months post-treatment, with target restoration to >50% of normal levels in GRN mutation carriers receiving AAV9-progranulin gene th |
| extraction_metadata | {'extraction_confidence': 0.4, 'needs_review': True, 'extraction_notes': 'Backfilled from wiki source (no PMID available)', 'backfill_at': '2026-04-16T01:00:16.900989'} |
🌍 Provenance Graph
9 nodes, 30 edges
derives from (16)
...and 11 more
Linked Artifacts (1168)