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. 2006 Nov 28;3(4):451–456. doi: 10.1016/j.ddstr.2006.10.008

Table 1.

Examples of neurological disorders and their respective molecular target(s) for RNAi

Neurological disorder Mutated protein(s)
Amyotrophic lateral sclerosis Cu/Zn superoxide dismutase
Spinocerebellar ataxia type 1 Ataxin-1
Huntington's disease Huntingtin
Spinobulbar muscular atrophy (Kennedy's disease) Androgen receptor
Familial Alzheimer's disease Amyloid precursor protein, presenilin 1 or 2
Parkinsonian frontotemporal dementia Tau
DYT1 dystonia TorsinA
Spinal muscular atrophy Survival motor neuron protein
Machado-Joseph disease MJD1
Prion-based disease Prion
Chronic pain Various ligand or ion-gated channels