tooluniverse-drug-target-validation

安装量: 113
排名: #7546

安装

npx skills add https://github.com/mims-harvard/tooluniverse --skill tooluniverse-drug-target-validation
Drug Target Validation Pipeline
Validate drug target hypotheses using multi-dimensional computational evidence before committing to wet-lab work. Produces a quantitative Target Validation Score (0-100) with priority tier classification and GO/NO-GO recommendation.
Key Principles
Report-first
- Create report file FIRST, then populate progressively
Target disambiguation FIRST
- Resolve all identifiers before analysis
Evidence grading
- Grade all evidence as T1 (experimental) to T4 (computational)
Disease-specific
- Tailor analysis to disease context when provided
Modality-aware
- Consider small molecule vs biologics tractability
Safety-first
- Prominently flag safety concerns early
Quantitative scoring
- Every dimension scored numerically (0-100 composite)
Negative results documented
- "No data" is data; empty sections are failures
Source references
- Every statement must cite tool/database
English-first queries
- Always use English terms in tool calls; respond in user's language
When to Use
Apply when users ask about:
"Is [target] a good drug target for [disease]?"
Target validation, druggability assessment, or target prioritization
Safety risks of modulating a target
Chemical starting points for target validation
GO/NO-GO recommendation for a target
Not for
(use other skills): general target biology (
tooluniverse-target-research
), drug compound profiling (
tooluniverse-drug-research
), variant interpretation (
tooluniverse-variant-interpretation
), disease research (
tooluniverse-disease-research
).
Input Parameters
Parameter
Required
Description
Example
target
Yes
Gene symbol, protein name, or UniProt ID
EGFR
,
P00533
disease
No
Disease/indication for context
Non-small cell lung cancer
modality
No
Preferred therapeutic modality
small molecule
,
antibody
,
PROTAC
Reference Files
SCORING_CRITERIA.md
- Detailed scoring matrices, evidence grading, priority tiers, score calculation
REPORT_TEMPLATE.md
- Full report template, completeness checklist, section format examples
TOOL_REFERENCE.md
- Verified tool parameters, known corrections, fallback chains, modality-specific guidance, phase-by-phase tool lists
QUICK_START.md
- Quick start guide
Scoring Overview
Total: 0-100 points
across 5 dimensions (details in SCORING_CRITERIA.md):
Dimension
Max
Sub-dimensions
Disease Association
30
Genetic (10) + Literature (10) + Pathway (10)
Druggability
25
Structure (10) + Chemical matter (10) + Target class (5)
Safety Profile
20
Expression (5) + Genetic validation (10) + ADRs (5)
Clinical Precedent
15
Based on highest clinical stage achieved
Validation Evidence
10
Functional studies (5) + Disease models (5)
Priority Tiers
80-100 = Tier 1 (GO) | 60-79 = Tier 2 (CONDITIONAL GO) | 40-59 = Tier 3 (CAUTION) | 0-39 = Tier 4 (NO-GO)
Evidence Grades
T1 (clinical proof) > T2 (functional studies) > T3 (associations) > T4 (predictions)
Pipeline Phases
Phase 0: Target Disambiguation (ALWAYS FIRST)
Resolve target to ALL identifiers before any analysis.
Steps
:
MyGene_query_genes
- Get initial IDs (Ensembl, UniProt, Entrez)
ensembl_lookup_gene
- Get versioned Ensembl ID (species="homo_sapiens" REQUIRED)
ensembl_get_xrefs
- Cross-references (HGNC, etc.)
OpenTargets_get_target_id_description_by_name
- Verify OT target
ChEMBL_search_targets
- Get ChEMBL target ID
UniProt_get_function_by_accession
- Function summary (returns list of strings)
UniProt_get_alternative_names_by_accession
- Collision detection
Output
Table of verified identifiers (Gene Symbol, Ensembl, UniProt, Entrez, ChEMBL, HGNC) plus protein function and target class.
Phase 1: Disease Association (0-30 pts)
Quantify target-disease association from genetic, literature, and pathway evidence.
Key tools
:
OpenTargets_get_diseases_phenotypes_by_target_ensembl
- Disease associations
OpenTargets_target_disease_evidence
- Detailed evidence (needs
efoId
+
ensemblId
)
OpenTargets_get_evidence_by_datasource
- Evidence by data source
gwas_get_snps_for_gene
/
gwas_search_studies
- GWAS evidence
gnomad_get_gene_constraints
- Genetic constraint (pLI, LOEUF)
PubMed_search_articles
- Literature (returns plain list of dicts)
OpenTargets_get_publications_by_target_ensemblID
- OT publications (uses
entityId
)
Phase 2: Druggability (0-25 pts)
Assess whether the target is amenable to therapeutic intervention.
Key tools
:
OpenTargets_get_target_tractability_by_ensemblID
- Tractability (SM, AB, PR, OC)
OpenTargets_get_target_classes_by_ensemblID
- Target classification
Pharos_get_target
- TDL: Tclin > Tchem > Tbio > Tdark
DGIdb_get_gene_druggability
- Druggability categories
alphafold_get_prediction
(param:
qualifier
) /
alphafold_get_summary
ProteinsPlus_predict_binding_sites
- Pocket detection
OpenTargets_get_chemical_probes_by_target_ensemblID
- Chemical probes
OpenTargets_get_target_enabling_packages_by_ensemblID
- TEPs
Phase 3: Chemical Matter (feeds Phase 2 scoring)
Identify existing chemical starting points for target validation.
Key tools
:
ChEMBL_search_targets
+
ChEMBL_get_target_activities
- Bioactivity data (note:
target_chembl_id__exact
with double underscore)
BindingDB_get_ligands_by_uniprot
- Binding data (affinity in nM)
PubChem_search_assays_by_target_gene
+
PubChem_get_assay_active_compounds
- HTS data
OpenTargets_get_associated_drugs_by_target_ensemblID
- Known drugs (
size
REQUIRED)
ChEMBL_search_mechanisms
- Drug mechanisms
DGIdb_get_gene_info
- Drug-gene interactions
Phase 4: Clinical Precedent (0-15 pts)
Assess clinical validation from approved drugs and clinical trials.
Key tools
:
FDA_get_mechanism_of_action_by_drug_name
/
FDA_get_indications_by_drug_name
drugbank_get_targets_by_drug_name_or_drugbank_id
(ALL params required:
query
,
case_sensitive
,
exact_match
,
limit
)
search_clinical_trials
(
query_term
REQUIRED)
OpenTargets_get_drug_warnings_by_chemblId
/
OpenTargets_get_drug_adverse_events_by_chemblId
Phase 5: Safety (0-20 pts)
Identify safety risks from expression, genetics, and known adverse events.
Key tools
:
OpenTargets_get_target_safety_profile_by_ensemblID
- Safety liabilities
GTEx_get_median_gene_expression
- Tissue expression (
operation="median"
REQUIRED)
HPA_search_genes_by_query
/
HPA_get_comprehensive_gene_details_by_ensembl_id
OpenTargets_get_biological_mouse_models_by_ensemblID
- KO phenotypes
FDA_get_adverse_reactions_by_drug_name
/
FDA_get_boxed_warning_info_by_drug_name
OpenTargets_get_target_homologues_by_ensemblID
- Paralog risks
Critical tissues to check
heart, liver, kidney, brain, bone marrow.
Phase 6: Pathway Context
Understand the target's role in biological networks and disease pathways.
Key tools
:
Reactome_map_uniprot_to_pathways
(param:
id
, NOT
uniprot_id
)
STRING_get_protein_interactions
(param:
protein_ids
as array,
species=9606
)
intact_get_interactions
- Experimental PPI
OpenTargets_get_target_gene_ontology_by_ensemblID
- GO terms
STRING_functional_enrichment
- Enrichment analysis
Assess
pathway redundancy, compensation risk, feedback loops.
Phase 7: Validation Evidence (0-10 pts)
Assess existing functional validation data.
Key tools
:
DepMap_get_gene_dependencies
- Essentiality (score < -0.5 = essential)
PubMed_search_articles
- Search for CRISPR/siRNA/knockout studies
CTD_get_gene_diseases
- Gene-disease associations
Phase 8: Structural Insights
Leverage structural biology for druggability and mechanism understanding.
Key tools
:
UniProt_get_entry_by_accession
- Extract PDB cross-references
get_protein_metadata_by_pdb_id
/
pdbe_get_entry_summary
/
pdbe_get_entry_quality
alphafold_get_prediction
/
alphafold_get_summary
- pLDDT confidence
ProteinsPlus_predict_binding_sites
- Druggable pockets
InterPro_get_protein_domains
/
InterPro_get_domain_details
- Domain architecture
Phase 9: Literature Deep Dive
Comprehensive collision-aware literature analysis.
Steps
:
Collision detection
Search
"{gene_symbol}"[Title]
in PubMed; if >20% off-topic, add filters (AND protein OR gene OR receptor)
Publication metrics
Total count, 5-year trend, drug-focused subset Key reviews : review[pt] filter in PubMed Citation metrics : openalex_search_works for impact data Broader coverage : EuropePMC_search_articles Phase 10: Validation Roadmap (Synthesis) Synthesize all phases into actionable output: Target Validation Score (0-100) with component breakdown Priority Tier (1-4) assignment GO/NO-GO Recommendation with justification Recommended Validation Experiments Tool Compounds for Testing Biomarker Strategy Key Risks and Mitigations Report Output Create file: [TARGET]_[DISEASE]_validation_report.md Use the full template from REPORT_TEMPLATE.md . Key sections: Executive Summary (score, tier, recommendation, key findings, critical risks) Validation Scorecard (all 12 sub-scores with evidence) Sections 1-14 covering each phase Completeness Checklist (mandatory before finalizing) Complete the Completeness Checklist (in REPORT_TEMPLATE.md) before finalizing to verify all phases were covered, all scores justified, and negative results documented.
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