SMA Research Platform

Targets

REFERENCE CATALOGUE

Genes, proteins, and pathways in the SMA Research Platform. This is a reference catalogue of ingested targets and their computational/literature evidence — inclusion is not a validation claim. Filter by role: SMA Targets covers the canonical SMA-axis primary list plus pathway-adjacent legacy targets. Off-Target Substrate shows the proteome-scan kinases, GPCRs, and ion channels used for selectivity screening. All Targets returns the complete catalogue. Role is stored on targets.metadata.target_role — all filtering happens server-side.

▶How does Target Role Classification work?

Every target row carries a target_role set by the backend classifier. Priority order on collisions:

primary_sma — canonical SMA-axis genes (SMN1/2, UBA1, PLS3, NCALD; NMJ: MuSK-AGRN-LRP4-DOK7-RAPSN-AChR; p38/MAPK11/MAPK14, JNK/MAPK8-10, KCNB1 (Kv2.1), KEAP1-NRF2, STMN2, SNPH, INPP5F, SARM1, and metabolic targets PGK1/PKM2 …). Note: the ROCK-LIMK-CFL axis is retained in the catalogue for completeness but is deprecated, not a primary therapeutic axis — mouse genetics (Bowerman 2010/2012) show no motor/innervation benefit. PERP was also dropped from the primary list: it is negative in the primary SMA literature (Šoltić & Fuller 2022, PMID 36419936, reports c-Fos rather than PERP upregulation in SMA), so it is not treated as a priority target.
sma_adjacent — Bryzant legacy research targets (ingested pre-2026-04-20, not on the primary list)
offtarget_substrate — proteome-scan kinases, GPCRs, ion channels used for Boltz-2 / Chai-1 selectivity screening
other — miscellaneous ingest without a clear flag

Classifier source: autonomous-jobs/scripts/classify_targets_role.sql. Filtering: /api/v2/targets?role=….

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