GenTIGSA Gene Database on Rare Genetic Disorders

Monogenic disorders list  

List of monogenic disorders and their associated genes
Monogenic disorders arise from mutations in a single gene, but variations within that gene or in multiple genes can also lead to similar phenotypes, typically following Mendelian inheritance patterns associated with specific genetic changes.

View information by inheritance mode


S.No.  Genetic Disorders
Modes of inheritance
Category Gene symbol Indian
context
 Links
81 Episodic ataxia 
Autosomal dominant
Neuromuscular disorders CACNB4 calcium voltage-gated channel auxiliary subunit beta 4
CACNA1A calcium voltage-gated channel subunit alpha1 A
SLC1A3 solute carrier family 1 member 3
KCNA1 potassium voltage-gated channel subfamily A member 1
SCN2A sodium voltage-gated channel alpha subunit 2
Reports
Updated as of Mar 09, 2023
PubMed
82 Facioscapulohumeral muscular dystrophy 
Autosomal dominant
Neuromuscular disorders SMCHD1 structural maintenance of chromosomes flexible hinge domain containing 1
DNMT3B DNA methyltransferase 3 beta
LRIF1 ligand dependent nuclear receptor interacting factor 1
Reports
Updated as of Mar 09, 2023
PubMed
83 Familial adenomatous polyposis 
Autosomal dominant, Autosomal recessive
Cancer disorders APC APC regulator of WNT signaling pathway
MUTYH mutY DNA glycosylase
MSH3 mutS homolog 3
NTHL1 nth like DNA glycosylase 1
STK11 serine/threonine kinase 11
Reports
Updated as of Nov 30, 2023
PubMed
84 Familial cancer of breast 
Autosomal dominant
Cancer disorders ATM ATM serine/threonine kinase
RAD51 RAD51 recombinase
BRCA2 BRCA2 DNA repair associated
BRCA1 BRCA1 DNA repair associated
PALB2 partner and localizer of BRCA2
BARD1 BRCA1 associated RING domain 1
CHEK2 checkpoint kinase 2
BRIP1 BRCA1 interacting helicase 1
PIK3CA phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha
CDH1 cadherin 1
PPM1D protein phosphatase, Mg2+/Mn2+ dependent 1D
TP53 tumor protein p53
AKT1 AKT serine/threonine kinase 1
RB1CC1 RB1 inducible coiled-coil 1
RAD51D RAD51 paralog D
PTEN phosphatase and tensin homolog
PTCH2 patched 2
RAD54L RAD54 like
ZNF462 zinc finger protein 462
Reports
Updated as of Feb 26, 2025
PubMed
85 Familial Cold Autoinflammatory Syndrome 
Autosomal dominant
Immune disorders NLRP12 NLR family pyrin domain containing 12
NLRP3 NLR family pyrin domain containing 3
PLCG2 phospholipase C gamma 2
NLRC4 NLR family CARD domain containing 4
Reports
Updated as of Sep 15, 2022
PubMed
86 Familial hypokalemia-hypomagnesemia 
Autosomal recessive
Nephrological disorders SLC12A3 solute carrier family 12 member 3
MMP2-AS1 MMP2 antisense RNA 1
Reports
Updated as of Jul 20, 2024
PubMed
87 Familial medullary thyroid carcinoma 
Autosomal dominant
Cancer disorders RET ret proto-oncogene
NTRK1 neurotrophic receptor tyrosine kinase 1
Reports
Updated as of Dec 29, 2023
PubMed
88 Fanconi anemia 
Autosomal recessive, X-linked dominant
Bone disorders FANCI FA complementation group I
PALB2 partner and localizer of BRCA2
FANCL FA complementation group L
FANCA FA complementation group A
BRIP1 BRCA1 interacting helicase 1
FANCF FA complementation group F
FANCG FA complementation group G
RAD51C RAD51 paralog C
FANCE FA complementation group E
BRCA2 BRCA2 DNA repair associated
FANCB FA complementation group B
FANCD2 FA complementation group D2
FANCC FA complementation group C
XRCC2 X-ray repair cross complementing 2
SLX4 SLX4 structure-specific endonuclease subunit
ERCC4 ERCC excision repair 4, endonuclease catalytic subunit
UBE2T ubiquitin conjugating enzyme E2 T
BRCA1 BRCA1 DNA repair associated
FANCM FA complementation group M
RAD51 RAD51 recombinase
MAD2L2 mitotic arrest deficient 2 like 2
RFWD3 ring finger and WD repeat domain 3
ZNF469 zinc finger protein 469
DCLRE1B DNA cross-link repair 1B
Reports
Updated as of Sep 05, 2023
PubMed
89 Fetal akinesia deformation sequence 1 
Autosomal recessive
Respiratory disorders MUSK muscle associated receptor tyrosine kinase
DOK7 docking protein 7
RAPSN receptor associated protein of the synapse
ASCC1 activating signal cointegrator 1 complex subunit 1
VPS13D vacuolar protein sorting 13 homolog D
ASPM assembly factor for spindle microtubules
CHRNG cholinergic receptor nicotinic gamma subunit
ASAH1 N-acylsphingosine amidohydrolase 1
ADSS1 adenylosuccinate synthase 1
EARS2 glutamyl-tRNA synthetase 2, mitochondrial
LGI4 leucine rich repeat LGI family member 4
RYR1 ryanodine receptor 1
GLDN gliomedin
Reports
Updated as of Feb 01, 2025
PubMed
90 Fetal akinesia deformation sequence 3 
Autosomal recessive
Respiratory disorders DOK7 docking protein 7
DOCK7 dedicator of cytokinesis 7
Reports
Updated as of Feb 01, 2025
PubMed