Inhibiting LINE-1 expression for ataxia

Inhibiting expression of the transposable element LINE-1 could help treat ataxia. Expression of LINE1 mRNA was higher in cerebellar samples from patients with ataxia than from healthy controls, and expression of genes that repress transposable elements was lower.

In a mouse model of ataxia caused by CRISPR-mediated LINE-1 activation in Purkinje cells, inhibiting LINE-1 gene expression in the cerebellum using the nucleoside reverse transcriptase inhibitor Epivir lamivudine decreased ataxia progression, interferon-stimulated gene expression, and hallmarks of astrogliosis and DNA damage compared with a non-nucleoside reverse transcriptase inhibitor that doesn't suppress LINE-1 activation...