Technology ID

Therapeutic Approaches to Inhibit Replication of ALS-related Endogenous Retroviruses

Lead Inventor
Nath, Avindra (NINDS)
Li, Wenxue (NINDS)
Lee, Myoung Hwa (NINDS)
Henderson, Lisa (NINDS)
Tyagi, Richa (NINDS)
Research Materials
Therapeutic Areas
Infectious Disease
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.
Commercial Applications
The use of existing retroviral drugs, or modified variants of these, could be useful to treat diseases related to HERV-K replication, including potentially ALS.

Competitive Advantages
Currently there are no available methods to block the replication of HERV-K.
Licensing Contact:
Dukhanina, Oksana
Phone: 301-594-6463