Trinucleotide Repeat Diseases

 

Polyglutamine (PolyQ) Diseases

Type

Gene

Normal/wildtype

Pathogenic

DRPLA (Dentatorubropallidoluysian atrophy

)

ATN1 or DRPLA

6 - 35

49 - 88

HD (Huntington's disease)

HTT (Huntingtin)

10 - 35

35+

SBMA (Spinobulbar muscular atrophy or Kennedy disease)

Androgen receptor on the X chromosome.

9 - 36

38 - 62

SCA1

ATXN1

6 - 35

49 - 88

SCA2

ATXN2

14 - 32

33 - 77

SCA3 (Machado-Joseph disease)

ATXN3

12 - 40

55 - 86

SCA6

CACNA1A

4 - 18

21 - 30

SCA7

ATXN7

7 - 17

38 - 120

SCA17

TBP

25 - 42

47 - 63

Non-Polyglutamine Diseases

Type

Gene

Codon

Normal/wildtype

Pathogenic

FRAXA (Fragile X syndrome)

FMR1, on the X-chromosome

CGG

6 - 53

230+

FRAXE (Fragile XE mental retardation)

AFF2 or FMR2, on the X-chromosome

GCC

6 - 35

200+

FRDA (Friedreich's ataxia)

FXN or X25, (frataxin)

GAA

7 - 34

100+

DM (Myotonic dystrophy)

DMPK

CTG

5 - 37

50+

SCA8 (Spinocerebellar ataxia Type 8)

OSCA or SCA8

CTG

16 - 37

110 - 250

SCA12 (Spinocerebellar ataxia Type 12)

PPP2R2B or SCA12

CAG On 5' end

7 - 28

66 - 78

Epidemiology

Pathogenesis

Clinical manifestations

Diagnosis

Treatment

Prognosis