Genes associated with “ataxia

539 genes foundHPO: AtaxiaOpen Targets: Ataxia36493 ClinVar P/LP variants5 PanelApp panels
How are genes scored? (0–100 composite)
-5–15
ClinGen
0–15
HPO Freq
0–15
Open Targets
0–12
Phen2Gene
0–10
ClinVar
0–8
Constraint
0–8
Dosage
0–8
OMIM CS
0–8
PanelApp
0–5
OMIM
0–5
G2P
0–4
Breadth
Tiers:Strong Candidates (≥20)Consider (≥8)Possible (≥3)
Evidence dots:
HPOClinVarPhen2GeneOpen TargetsPanelApp
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Strong Candidates

49 genes
1
ITPR1

inositol 1,4,5-trisphosphate receptor type 1

62
score
ClinGen: DefinitivePanel: Green (4)P2G #27GTR ↑

spinocerebellar ataxia type 29

Frequency
100%
n=7
P/LP Variants
119
OT Score
0.14
2
EBF3

EBF transcription factor 3

58
score
ClinGen: DefinitivePanel: Green (3)GTR ↑

hypotonia, ataxia, and delayed development syndrome

Frequency
71%
n=7
P/LP Variants
76
OT Score
0.43
3
CACNA1A

calcium voltage-gated channel subunit alpha1 A

54
score

spinocerebellar ataxia type 6

Frequency
20%
n=5
P/LP Variants
9
OT Score
0.34
4
ATM

ATM serine/threonine kinase

53
score
ClinGen: DefinitivePanel: Green (5)P2G #32GTR ↑
Frequency
-
P/LP Variants
34
OT Score
0.13
37
score

Machado-Joseph disease

Frequency
100%
n=57
P/LP Variants
4
OT Score
-
37
score

spinocerebellar ataxia type 11

Frequency
100%
n=18
P/LP Variants
15
OT Score
-
36
score
ClinGen: DefinitivePanel: Green (4)GTR ↑
Frequency
-
P/LP Variants
1
OT Score
-
36
score
ClinGen: DefinitivePanel: Green (4)GTR ↑
Frequency
-
P/LP Variants
1
OT Score
-
36
score
ClinGen: DefinitivePanel: Green (4)GTR ↑

spinocerebellar ataxia type 13

Frequency
100%
n=12
P/LP Variants
-
OT Score
-
34SYNE1
DefP:G×4
34SETX
DefP:G×4

senataxin

34KCNA1
DefP:G×4
31RNF220
P:G×2

leukodystrophy, hypomyelinating, 23, with ataxia, deafness, liver dysfunction, and dilated cardiomyopathy

31FXN
DefP:G×4
31DAGLA
P:G×3

diacylglycerol lipase alpha

31KCND3
DisP:G×4

spinocerebellar ataxia type 19/22

31CACNA1G
P:G×4

spinocerebellar ataxia type 42

30DARS2
DefP:G×4

aspartyl-tRNA synthetase 2, mitochondrial

30DNMT1
DefP:G×4

autosomal dominant cerebellar ataxia, deafness and narcolepsy

30TUBB4A
DefP:G×4

hypomyelinating leukodystrophy 6

30POLG
LimP:G×4

progressive external ophthalmoplegia with mitochondrial DNA deletions, autosomal recessive 1

30SPG7
P:G×4

hereditary spastic paraplegia 7

29GEMIN5
DefP:G×2

neurodevelopmental disorder with cerebellar atrophy and motor dysfunction

29NPC1
DefP:G×4
29ATXN2
DefP:R×4

spinocerebellar ataxia type 2

29PITRM1
P:G×2

spinocerebellar ataxia, autosomal recessive 30

28PRICKLE1
LimP:R×4
28KIF1C
P:G×4

spastic ataxia 2

27SLC9A6
DefP:G×4

neurodegenerative disorder, X-linked, female-restricted, with parkinsonism and cognitive impairment

27SPTBN2
DefP:G×4

autosomal recessive spinocerebellar ataxia 14

27ASL
DefP:G×2#10

argininosuccinate lyase

27PLP1
Def#15

Pelizeaus-Merzbacher spectrum disorder

26ALDH5A1
DefP:G×2#18

succinic semialdehyde dehydrogenase deficiency

26PNPLA6
DefP:G×4

ataxia-hypogonadism-choroidal dystrophy syndrome

26ATP1A3
DefP:G×4

cerebellar ataxia-areflexia-pes cavus-optic atrophy-sensorineural hearing loss syndrome

26INPP5E
DefP:G#3

Joubert syndrome 1

25ATG7
StrP:G×2

autophagy related 7

24ANO10
DefP:G×4

autosomal recessive spinocerebellar ataxia 10

24FA2H
DefP:G×2
23PEX16
DefP:G×4

peroxisome biogenesis disorder 8B

23MECP2
Def

syndromic X-linked intellectual disability Lubs type

23WWOX
DefP:G×4
23KCNJ10
DefP:G×4

EAST syndrome

22PEX6
DefP:G×3
22TMEM237
DefP:G#1

Joubert syndrome 14

22CYP27A1
DefP:G×4#14

cerebrotendinous xanthomatosis

21CA8
ModP:G×4

cerebellar ataxia, intellectual disability, and dysequilibrium syndrome 3

21GRM1
P:G×4

autosomal recessive spinocerebellar ataxia 13

20AFG3L2
DefP:G×4

spastic ataxia 5

Consider

138 genes
20AHI1
DefP:G

Joubert syndrome 3

19ARL13B
DefP:G

Joubert syndrome 8

19SLC52A2
DefP:G×3

Brown-Vialetto-van Laere syndrome 2

19ATP1A2
DefP:G
19TTR
DefSF
19GCH1
Def

dystonia 5

18SPG11
Def
18OPA1
DefP:G×3

Behr syndrome

17LIG3
DefP:A×2

DNA ligase 3

17SIL1
DefP:G×4

Marinesco-Sjogren syndrome

17HEXB
DefP:G×4

Sandhoff disease

17DLG4
DefP:G×2
17ERCC4
DefP:G
17PHGDH
DefP:A×2
17PRKCG
P:G×4

spinocerebellar ataxia type 14

17UBE3A
Def#13
16SLC19A3
Def
16VPS13D
LimP:G×4
16MTPAP
DefP:A×4

spastic ataxia 4

16GMPPA
DefP:G

alacrima, achalasia, and intellectual disability syndrome

16DNAJC19
DefP:G×4
16GBA2
DefP:G×4
16NHLRC1
DefP:G×4
16PLA2G6
DefP:G×4
16SCN8A
DefP:G×4
16COX10
Def#6

mitochondrial complex IV deficiency, nuclear type 3

16ATCAY
P:G×4

Cayman type cerebellar ataxia

16VPS41
P:G×3

VPS41 subunit of HOPS complex

15HMBS
DefP:G×2

leukoencephalopathy, porphyria-related

15PMM2
DefP:G
15PEX7
Def
15FDXR
DefP:G×2

multiple mitochondrial dysfunctions syndrome 9b

15MRE11
RefP:G×4

ataxia-telangiectasia-like disorder 1

15GPAA1
StrP:G×4

glycosylphosphatidylinositol biosynthesis defect 15

15APOB
DefSF
15VHL
DefSF
15WDR45
Def
15PIBF1
ModP:G

progesterone immunomodulatory binding factor 1

15CACNB4
RefP:R×4

episodic ataxia type 5

15TGM6
P:G×4

spinocerebellar ataxia type 35

14NEU1
DefP:A×3

neuraminidase 1

14FGF14
P:G×4

spinocerebellar ataxia 27A

14ABCB7
ModP:G×4

X-linked sideroblastic anemia with ataxia

14NFIX
Def
14DLD
Def
14ELOVL5
P:A×4

spinocerebellar ataxia type 38

14TCF4
Def

Pitt-Hopkins syndrome

14AP1S2
DefP:G×4

syndromic X-linked intellectual disability 5

14OPHN1
DefP:G×4

X-linked intellectual disability-cerebellar hypoplasia syndrome

14COQ4
DefP:G×3

neonatal encephalomyopathy-cardiomyopathy-respiratory distress syndrome

14CSTB
DefP:G×4

Unverricht-Lundborg syndrome

14COQ8A
P:G×4

coenzyme Q8A

14ATXN1
P:R×4

spinocerebellar ataxia type 1

14DYNC1H1
DefP:A×4

dynein cytoplasmic 1 heavy chain 1

14KCNA2
DefP:G×3

developmental and epileptic encephalopathy, 32

14WFS1
DefP:G×4

Wolfram syndrome 1

14CACNA2D2
ModP:G

calcium voltage-gated channel auxiliary subunit alpha2delta 2

14FEM1C
P:G×2

fem-1 homolog C

13AP2M1
Def

intellectual developmental disorder 60 with seizures

13STUB1
P:G×4

spinocerebellar ataxia 48

13ADA2
Def
13AHDC1
Def
13CHCHD10
Def

CHARCOT-MARIE-TOOTH DISEASE, X-LINKED RECESSIVE, 4, WITH OR WITHOUT CEREBELLAR ATAXIA; CMTX4

13EA1

EPISODIC ATAXIA, TYPE 1; EA1

13EXOSC3
P:G×4
13FMR1
DefP:R×4

fragile X-associated tremor/ataxia syndrome

13GNE
DefP:G
13GRIN2A
Def
13IFT74
DefP:G
13KCNC1
Def
13LYST
Def
13PIGO
DefP:G

SPINOCEREBELLAR ATAXIA 1; SCA1

SPINOCEREBELLAR ATAXIA 7; SCA7

SPINOCEREBELLAR ATAXIA, X-LINKED 1; SCAX1

Spinocerebellar ataxia, X-linked 5

SPINOCEREBELLAR ATAXIA, X-LINKED 6, WITH OR WITHOUT SIDEROBLASTIC ANEMIA; SCAX6

13CUL4B
Def

X-linked intellectual disability, Cabezas type

12PIGS
DefP:G

glycosylphosphatidylinositol biosynthesis defect 18

12PUM1
P:G
12IFT140
Def
12TBC1D24
Def
12MTFMT
DefP:G×2

mitochondrial complex I deficiency, nuclear type 27

12SCO2
Def

cardioencephalomyopathy, fatal infantile, due to cytochrome c oxidase deficiency 1

12NDUFS7
Def

mitochondrial complex I deficiency, nuclear type 3

12ATXN7
P:R×4
12MSTO1
DefP:G×3

mitochondrial myopathy-cerebellar ataxia-pigmentary retinopathy syndrome

12TANGO2
P:G×2
12PNP
Def

purine nucleoside phosphorylase deficiency

12GRID2
P:G×4

autosomal recessive spinocerebellar ataxia 18

12NDUFAF6
Def

mitochondrial complex I deficiency, nuclear type 17

12MTAP
Mod

methylthioadenosine phosphorylase

12NARS1
Mod

neurodevelopmental disorder with microcephaly, impaired language, epilepsy, and gait abnormalities

11LARS2
DefP:G×2

Perrault syndrome 4

11MKS1
DefP:G

Joubert syndrome 28

11BBS1
DefP:G×2

Bardet-Biedl syndrome 1

11CLN5
DefP:G×2

neuronal ceroid lipofuscinosis 5

11KIF1A
DefP:G×2

Dysmetria

11PEX5
Def
11ERCC6
Def

de Sanctis-Cacchione syndrome

11PI4KA
P:G×3

gastrointestinal defects and immunodeficiency syndrome 2

10RUBCN
P:R×4

autosomal recessive spinocerebellar ataxia 15

10CLCN2
RefP:G×4

leukoencephalopathy with mild cerebellar ataxia and white matter edema

10COQ2
Def

coenzyme Q10 deficiency, primary, 1

10DPYSL5
P:G×2

Ritscher-Schinzel syndrome 4

10B4GALT1
P:G
10TBR1
Def

T-box brain transcription factor 1

10GRIN2B
Def

glutamate ionotropic receptor NMDA type subunit 2B

10GLB1
Def

GM1 gangliosidosis type 3

10LETM1
P:G×2

neurodegeneration, childhood-onset, with multisystem involvement due to mitochondrial dysfunction

10FTL
Def

neuroferritinopathy

9MAN2B1
Def
9MOCS1
Def
9NBN
Def
9SUOX
Def
9ARX
Def
9CDKN2A
Def
9EEF2
P:A×4

spinocerebellar ataxia type 26

9NFASC
P:G×3

neurodevelopmental disorder with central and peripheral motor dysfunction

9NGLY1
DefP:G

congenital disorder of deglycosylation 1

9TBC1D23
P:G×3

pontocerebellar hypoplasia, type 11

9WDR81
P:G×4

cerebellar ataxia, intellectual disability, and dysequilibrium syndrome 2

intellectual disability-sparse hair-brachydactyly syndrome

9CAPN1
P:G

autosomal recessive spastic paraplegia type 76

9PRKACG
Dis
9LMNB1
Def

leukodystrophy, demyelinating, adult-onset, autosomal dominant, typical

9EXOSC5
P:A×2

exosome component 5

9SCN1B
Def

developmental and epileptic encephalopathy, 52

mitochondrial complex I deficiency, nuclear type 28

8GPHN
Mod
8SOX9
Def

SRY-box transcription factor 9

8SOX10
Def

PCWH syndrome

8GRIK2
Def

glutamate ionotropic receptor kainate type subunit 2

8BEAN1
P:R×4

spinocerebellar ataxia type 31

8NOP56
P:R×4

spinocerebellar ataxia type 36

Possible

327 genes — click to expand
8SCARB2
Def

action myoclonus-renal failure syndrome

8NEMF
Str

nuclear export mediator factor

7ARL3
P:A

Joubert syndrome 35

7DYRK1A
Def

DYRK1A-related intellectual disability syndrome

7KARS1
Lim

lysyl-tRNA synthetase 1

7DNAI1
Def
7DOCK8
Def
7EPG5
Def
7FANCG
Def
7GAN
Def
7GLDC
Def
7IL11RA
Def
7KCNV2
Def
7KCTD7
Def
7MLC1
Def
7NFIB
Def
7PAX5
Def
7RFX3
Def
7RMRP
Def
7TEK
Def
7TOPORS
Def
7TPM2
Def
7TRPM3
Def
7VCP
Def
7PRX
Def

periaxin

calcium/calmodulin dependent protein kinase IV

mitochondrially encoded ATP synthase membrane subunit 6

7MME
DefP:R

spinocerebellar ataxia 43

SKI family transcriptional corepressor 2

6HARS1
Ref

histidyl-tRNA synthetase 1

6RUSC2
Mod

Meckel syndrome 13

cortical dysplasia-focal epilepsy syndrome

6FAT2
P:A

spinocerebellar ataxia 45

5FGF12
Def

developmental and epileptic encephalopathy, 47

5PCNA
Lim

ataxia-telangiectasia-like disorder 2

ATAXIA-PANCYTOPENIA SYNDROME; ATXPC

CEREBELLAR ATAXIA, IMPAIRED INTELLECTUAL DEVELOPMENT, AND DYSEQUILIBRIUM SYNDROME 1; CAMRQ1

5CLPB
Lim

EPISODIC ATAXIA, TYPE 2; EA2

SPINOCEREBELLAR ATAXIA, AUTOSOMAL RECESSIVE, WITH AXONAL NEUROPATHY 2; SCAN2

SPINOCEREBELLAR ATAXIA, AUTOSOMAL RECESSIVE 34; SCAR34

5SLC1A1
Lim

SPASTIC ATAXIA 7, AUTOSOMAL DOMINANT; SPAX7

5TJP2
Lim
5ERBB4
Lim

erb-b2 receptor tyrosine kinase 4

5CAV1
Def

partial lipodystrophy, congenital cataracts, and neurodegeneration syndrome

spastic paraplegia-severe developmental delay-epilepsy syndrome

5THAP11
Lim

spinocerebellar ataxia 51

5TBCE
Mod

encephalopathy, progressive, with amyotrophy and optic atrophy

hereditary spastic paraplegia 9A

5ACOX2
Def

congenital bile acid synthesis defect 6

5MMADHC
Def

homocystinuria-megaloblastic anemia cblD type

5NONO
Def

syndromic X-linked intellectual disability 34

5NSUN2
Def

intellectual disability, autosomal recessive 5

5PRF1
Def

familial hemophagocytic lymphohistiocytosis 2

heat shock protein 90 beta family member 1

4CDC42
Def

macrothrombocytopenia-lymphedema-developmental delay-facial dysmorphism-camptodactyly syndrome

4SGPL1
Def

nephrotic syndrome 14

DnaJ heat shock protein family (Hsp40) member A2

CD5 molecule like

4ZFHX3
P:R×2

spinocerebellar ataxia type 4

4COQ5
Mod

coenzyme q10 deficiency, primary, 9

KRAB domain containing 3

4CERS1
Mod

progressive myoclonic epilepsy type 8

4ACADS
Def

short chain acyl-CoA dehydrogenase deficiency

family with sequence similarity 210 member B

kinase suppressor of ras 1

T-box transcription factor 15

3AMACR
Def

alpha-methylacyl-CoA racemase deficiency

3AP4M1
Def

hereditary spastic paraplegia 50

3BOLA3
Def

multiple mitochondrial dysfunctions syndrome 2

neurodegeneration with brain iron accumulation 4

3CTC1
Def

cerebroretinal microangiopathy with calcifications and cysts 1

3ERCC5
Def

xeroderma pigmentosum group G

3ETHE1
Def

ethylmalonic encephalopathy

3NANS
Def

spondyloepimetaphyseal dysplasia, Genevieve type

Hartnup disease

3TCN2
Def

transcobalamin II deficiency

Data aggregated from HPO/Monarch, ClinVar, OMIM (phenotypes + clinical synopsis), Open Targets, PanelApp, Phen2Gene, and Gene2Phenotype. Click a gene to view full details.