HIV-1 RNA target site for the development of HIV drug or gene therapy


Published: 4Oct2019

Invention 14004

HIV-1 RNA target site for the development of HIV drug or gene therapy

A highly conserved HIV-1 RNA sequence that could be used as a target site for potential gene therapy treatments has been identified at McGill University.

Market Need

The current HIV treatment regimen of chemotherapy and antiretroviral therapy only suppresses the disease, requiring continued lifelong treatment. Directed gene therapy, on the other hand, is an approach that alters the genetic code of cells or viruses. One promising form of gene therapy, RNA interference (RNAi), binds to a specific RNA sequence in order to suppress that gene’s function. With the proper target site, short interfering RNA (siRNA) or short hairpin RNA (shRNA) treatment could either be combined with other drugs or as a stand-alone way of blocking entry to human cells. While only a few gene therapy products have been FDA approved, early HIV gene therapy products are joining hundreds of promising cancer and neurodegenerative disorder clinical studies.

Technology Summary

In this invention, the HIV-1 RNA target site identified by the Gatignol Lab is highly conserved and easily accessible to RNA molecules. For RNAi inhibitory targeting to be successful in HIV, the target sequence needs to be highly conserved across multiple HIV-1 strains. RNAi treatments would therefore be able to bind to a greater percentage of HIV strains as this region has a lower rate of mutation and is therefore more likely to be present in HIV+ individuals. Since this is a more highly conserved sequence than other patented sequences, the inventors are confident a useful treatment can be produced from this target site. The investigators have importantly shown that this target site is also capable of being used with combinatorial treatment strategies of antisense, siRNA, and shRNA, although ribozyme or aptamer accessibility has not been screened.


  • Superior to many other patented sites in terms of gene conservation across HIV-1 strains, specificity, and accessibility to different small RNA technologies
  • Can be used in drug or gene therapy to treat HIV infection using antisense RNA, siRNA, shRNA
  • Higher conservation means a lower chance of mutation and presence in higher proportion of different HIV-1 strains

Patent Status

Issued US, filed CA


Contact Information

Mostafa Ghozlan
Innovation and Partnerships
mostafa.ghozlan [at]
Back to top