Therapeutic Approaches to Inhibit Replication of ALS-related Endogenous Retroviruses

The technology relates to therapeutic approaches that inhibit and block the replication of the endogenous HERV-K retrovirus. Previous work has shown that patients with Amyotrophic Lateral Sclerosis (ALS) can have HERV-K activation. In animal models, activation of HERV-K can lead to neurodegenerative symptoms similar to those exhibited by ALS patients. Work in these animal models has allowed the identification of the responsible transcription factor (TDP-43) as well as the corresponding positions of the HERV-K promoter binding sites.

This work has shown that existing retroviral drugs used for treating HIV infection can also inhibit HERV-K replication, although this requires a different dose range and combination of drugs. In addition, antisense molecules have been developed that can block the replication of HERV-K specifically.