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
181 Neurodegeneration with brain iron accumulation 
Autosomal dominant, Autosomal recessive
Neurodegenerative disorders PLA2G6 phospholipase A2 group VI
C19orf12 chromosome 19 open reading frame 12
WDR45 WD repeat domain 45
COASY Coenzyme A synthase
FA2H fatty acid 2-hydroxylase
CRAT carnitine O-acetyltransferase
REPS1 RALBP1 associated Eps domain containing 1
ATP13A2 ATPase cation transporting 13A2
DCAF17 DDB1 and CUL4 associated factor 17
FTH1 ferritin heavy chain 1
Reports
Updated as of Feb 01, 2025
PubMed
182 Neuronal ceroid lipofuscinosis 
Autosomal dominant, Autosomal recessive
Metabolic disorders/Lysosomal storage disorders MFSD8 major facilitator superfamily domain containing 8
CLN5 CLN5 intracellular trafficking protein
TPP1 tripeptidyl peptidase 1
CLN8 CLN8 transmembrane ER and ERGIC protein
CLN3 CLN3 lysosomal/endosomal transmembrane protein, battenin
PPT1 palmitoyl-protein thioesterase 1
CTSD cathepsin D
CTSF cathepsin F
CLN6 CLN6 transmembrane ER protein
GRN granulin precursor
DNAJC5 DnaJ heat shock protein family (Hsp40) member C5
KCTD7 potassium channel tetramerization domain containing 7
Reports
Updated as of Sep 05, 2023
PubMed
183 Non-ketotic hyperglycinemia 
Autosomal recessive
Metabolic disorders AMT aminomethyltransferase
GLDC glycine decarboxylase
GCSH glycine cleavage system protein H
PCDH19 protocadherin 19
Reports
Updated as of Feb 20, 2024
PubMed
184 Non-small cell lung carcinoma 
Cancer disorders BRAF B-Raf proto-oncogene, serine/threonine kinase
IRF1 interferon regulatory factor 1
ERBB2 erb-b2 receptor tyrosine kinase 2
KRAS KRAS proto-oncogene, GTPase
EGFR epidermal growth factor receptor
PIK3CA phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha
NRAS NRAS proto-oncogene, GTPase
DDR2 discoidin domain receptor tyrosine kinase 2
MAP2K1 mitogen-activated protein kinase kinase 1
ALK ALK receptor tyrosine kinase
ARAF A-Raf proto-oncogene, serine/threonine kinase
STK11 serine/threonine kinase 11
NOTCH1 notch receptor 1
Reports
Updated as of Dec 29, 2023
PubMed
185 Noonan syndrome 
Autosomal dominant
Neurodevelopmental disorders SHOC2 SHOC2 leucine rich repeat scaffold protein
RRAS2 RAS related 2
KRAS KRAS proto-oncogene, GTPase
SOS1 SOS Ras/Rac guanine nucleotide exchange factor 1
PTPN11 protein tyrosine phosphatase non-receptor type 11
CBL Cbl proto-oncogene
NRAS NRAS proto-oncogene, GTPase
RAF1 Raf-1 proto-oncogene, serine/threonine kinase
BRAF B-Raf proto-oncogene, serine/threonine kinase
RIT1 Ras like without CAAX 1
SPRED1 sprouty related EVH1 domain containing 1
LZTR1 leucine zipper like post translational regulator 1
SOS2 SOS Ras/Rho guanine nucleotide exchange factor 2
PPP1CB protein phosphatase 1 catalytic subunit beta
MAP2K1 mitogen-activated protein kinase kinase 1
A2ML1 alpha-2-macroglobulin like 1
RRAS RAS related
MRAS muscle RAS oncogene homolog
GJB2 gap junction protein beta 2
MAPK1 mitogen-activated protein kinase 1
CLTC clathrin heavy chain
SPRED2 sprouty related EVH1 domain containing 2
MAP2K2 mitogen-activated protein kinase kinase 2
RASA2 RAS p21 protein activator 2
Reports
Updated as of Mar 09, 2023
PubMed
186 Obsessive compulsive disorder 
Autosomal dominant
Neurodevelopmental disorders HTR2A 5-hydroxytryptamine receptor 2A
SLC6A4 solute carrier family 6 member 4
HTR2C 5-hydroxytryptamine receptor 2C
Reports
Updated as of Mar 09, 2023
PubMed
187 Oculopharyngeal muscular dystrophy 
Autosomal dominant, Autosomal recessive
Neuromuscular disorders PABPN1 poly(A) binding protein nuclear 1
ANXA11 annexin A11
HNRNPA2B1 heterogeneous nuclear ribonucleoprotein A2/B1
Reports
Updated as of Nov 30, 2023
PubMed
188 Oromandibular-limb hypogenesis spectrum 
Autosomal dominant
Neurodevelopmental disorders SIM1 SIM bHLH transcription factor 1
EBF3 EBF transcription factor 3
CHN1 chimerin 1
Reports
Updated as of Sep 15, 2022
PubMed
189 Osteogenesis imperfecta 
Autosomal dominant, Autosomal recessive, X-linked dominant
Bone disorders P3H1 prolyl 3-hydroxylase 1
FKBP10 FKBP prolyl isomerase 10
CRTAP cartilage associated protein
PPIB peptidylprolyl isomerase B
COL1A2 collagen type I alpha 2 chain
COL1A1 collagen type I alpha 1 chain
SERPINH1 serpin family H member 1
SERPINF1 serpin family F member 1
BMP1 bone morphogenetic protein 1
TMEM38B transmembrane protein 38B
WNT1 Wnt family member 1
IFITM5 interferon induced transmembrane protein 5
SP7 Sp7 transcription factor
SPARC secreted protein acidic and cysteine rich
TENT5A terminal nucleotidyltransferase 5A
MBTPS2 membrane bound transcription factor peptidase, site 2
CREB3L1 cAMP responsive element binding protein 3 like 1
SLC34A1 solute carrier family 34 member 1
MESD mesoderm development LRP chaperone
KDELR2 KDEL endoplasmic reticulum protein retention receptor 2
GBE1 1,4-alpha-glucan branching enzyme 1
LRP5 LDL receptor related protein 5
XYLT2 xylosyltransferase 2
PHLDB1 pleckstrin homology like domain family B member 1
Reports
Updated as of Sep 15, 2022
PubMed
190 Otospondylomegaepiphyseal dysplasia 
Autosomal recessive
Bone disorders COL11A2 collagen type XI alpha 2 chain
COL2A1 collagen type II alpha 1 chain
Reports
Updated as of Jan 04, 2024
PubMed