Research ArticleALS

Human endogenous retrovirus-K contributes to motor neuron disease

See allHide authors and affiliations

Science Translational Medicine  30 Sep 2015:
Vol. 7, Issue 307, pp. 307ra153
DOI: 10.1126/scitranslmed.aac8201

A viral endgame

A large number of viral sequences are present in the human genome but remain silent. However, under pathological conditions, these viruses can get expressed. Li et al. now report that one such virus, human endogenous retrovirus-K, is expressed in neurons of a subpopulation of patients with amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease. The envelope protein of this virus causes degeneration of neurons, and transgenic animals expressing this protein develop an ALS-like syndrome caused by nucleolar dysfunction in motor neurons. Reactivation of the virus is regulated by the transcription factor TDP-43. Thus, therapeutic approaches against this virus could potentially alter the course of the disease.

View Full Text

Stay Connected to Science Translational Medicine