IMAGE: Huntington’s disease is due to a fault (a repeat of DNA) on chromosome 4. This repetition of genetic information results in the production of an abnormal protein called huntingtin,. Mutant huntingtin aggregates in clusters in the nuclei of certain neurons in the brain, particularly the striatum, a brain structure involved in controlling movement. These neurons degenerate and eventually die, explaining the appearance of abnormal movements. Then huntingtin accumulates in the nuclei of neurons in the cerebral cortex also causing neuronal degeneration with consequent cognitive impairment (memory, attention …) and psychiatric disorders.
A drug that would be the first to target the cause of Huntington’s disease (HD) is effective and safe when tested in mice and monkeys...
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