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. Author manuscript; available in PMC: 2012 Dec 10.
Published in final edited form as: Nat Rev Drug Discov. 2011 Dec 1;10(12):930–944. doi: 10.1038/nrd3453

Table 1.

Neurodegenerative diseases that are associated with protein misfolding

Neurodegenerative
diseases
Aggregation-
prone protein
Neuronal
population lost
Disease
symptoms
Current
therapies
Refs
Polyglutamine
disorders: Huntington’s disease,
spinal and bulbar
muscular atrophy,
spinocerebellar
ataxias and others
Polyglutamine
tracts within
distinct
pathogenic
proteins
Variable Chorea, cognitive decline,
depression
and anxiety,
dementia and
ataxia
Tetrabenazine,
amantadine,
remacemide,
antipsychotics
and
antidepressants
13
Parkinson’s disease α-synuclein Substantia nigra
pars compacta,
ventral tegmental
area, autonomic
ganglia and
neurons of the
myenteric plexus
Tremors,
bradykinesia,
postural instability,
rigidity, dementia,
depression, anxiety,
gastrointestinal
dysfunction and
hallucinations
Levodopa/
carbidopa,
dopamine agonists
and monoamine
oxidase inhibitors
7,8
Alzheimer’s disease Amyloid-β,
hyperphosphory-
lated tau
Cortical and
subcortical
neurons, locus
coeruleus and
cholinergic
neurons
Learning
and memory
impairments,
motor
dysfunction,
and irritability
Acetylcholin
esterase inhibitors,
memantine and
antipsychotics
9,10
Amyotrophic
lateral sclerosis/
Lou Gehrig’s
disease
Superoxide
dismutase, TAR DNA
binding protein 43
and others
Motor neurons of
the cortex, brain
stem and spinal
cord
Muscular atrophy
and motor
dysfunction
Riluzole 11
Prion disorders:
Creutzfeldt–Jakob
disease, Gerstmann–
Straussler syndrome,
fatal familial
insomnia and kuru
Various forms
of the prion
protein such as
scrapie (PrPSc)
Variable Spongiform
encephalopathy,
personality
changes,
depression and
insomnia
None 12