Almost half of our genome is occupied by virus-like elements called “retrotransposons”, which have been introduced into our genome over millions of years. Retrotransposons are mobile DNA elements that use a “reverse transcriptase” enzyme to self-replicate and proliferate. Since these activities can be damaging to our cells, our bodies maintain strict control over retrotransposon expression.

Today, the only retrotransposons in our genome that are capable of becoming active are human-specific LINE-1 elements. Abnormal proteins that lead to neurodegenerative diseases, such as tau and TDP-43, can increase the expression of these LINE-1 elements, initiating a cascade of events in the neuron that leads to its dysfunction and death.

Transposon Therapeutics, Inc. is developing inhibitors of LINE-1 reverse transcriptase and other mediators of LINE-1 pathology for the treatment of neurodegenerative disease.

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