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Molecules of Clinical Relevance

Name the following molecules which are targeted by medications, and also mutated in genetic diseases and/or targeted by autoantibodies in autoimmune conditions.
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Last updated: March 13, 2026
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First submittedMarch 13, 2026
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Average score100.0%
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Autoantibodies in ...
Inherited mutations in ...
Molecule
Targeted by ...
Autoimmune thyroiditis
-
Thyroid peroxidase (TPO)
Methimazole, propylthiouracil (PTU)
Anti-____ encephalitis
GRIN disorders
NMDA-R
Ketamine, memantine
Anti-_______ immune-mediated necrotizing myopathy (IMNM)
Limb-girdle muscular dystrophy (LGMD)
HMG-CoA reductase (HMGCR)
Statins
Immune thrombocytopenia purpura (ITP)
Glanzmann's thrombasthenia
Glycoprotein IIb/IIIa (GPIIb/IIIa)
Abciximab, eptifibatide, tirofiban
-
X-linked agammaglobulinemia (XLA)
Bruton's tyrosine kinase (BTK)
Ibrutinib, acalabrutinib, zanubrutinib
Acquired Gitelman syndrome
Gitelman syndrome
Sodium-chloride cotransporter (NCC / NCCT)
Hydrochlorothiazide, chlorthalidone, metolazone
-
Bartter syndrome
Sodium-potassium-chloride cotransporter (NKCC)
Furosemide, bumetanide
-
Liddle's syndrome, pseudohypoaldosteronism type I
Epithelial sodium channel (ENaC)
Amiloride, triamterene
Anti-______ systemic sclerosis (SSc)
-
Topoisomerase I (Scl-70)
Irinotecan, topotecan
Myasthenia gravis (MG)
Congenital myasthenic syndrome (CMS)
Nicotinic acetylcholine receptor (nAChR)
Rocuronium, succinylcholine (suxamethonium)
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