Examples of approved drugs with causal support from tissue-specific human autoimmunity or human genetics. SOST, sclerostin; IL6R, interleukin 6 receptor; PCSK9, proprotein convertase subtilisin/kexin type 9; GLP1R, glucagon-like peptide–1 receptor; IL23A, interleukin 23α; IL12B, interleukin 12β.
| Clinical disorder | Tissue-specific autoimmunity | Drug |
| Encephalitis | DRD2 | Antipsychotics |
| Narcolepsy | Orexin neurons | Suvorexant |
| Diabetes | Pancreatic beta cells | Insulin |
| Myasthenia gravis | Acetylcholine receptors | Acetylcholinesterase inhibitors |
| Thrombotic thrombocytopenic purpura | ADAMTS13 on platelets | Caplacizumab* |
| Clinical disorder | Gene(s) | Drug(s) |
| Schizophrenia | DRD2 | Antipsychotics |
| Sclerosteosis and van Buchem disease | SOST | Romosozumab* |
| Rheumatoid arthritis | IL6R | Tocilizumab |
| High cholesterol | PCSK9 | Evolocumab and alirocumab |
| Diabetes | GLP1R | Incretin mimetics |
| Psoriasis | IL23A, IL23R, and IL12B | Ustekinumab |
| Atopic dermatitis | IL4RA | Dupilumab* |
*Not yet approved.