Gene Therapy Vector for Modification for anti HIV Gene Therapy

Technology #d-1097

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Dr. Himanshu Garg
Dr. Garg's long standing interest has been in studying the role of HIV Env glycoprotein and more specifically the fusogenic activity of the Env glycoprotein and how it determines HIV pathogenesis. He previously demonstrated that hemifusion mediated by HIV gp41 is a prime mediator of bystander apoptosis and that some gp41 mutations that are seen in HIV infected patients undergoing Enfuvirtide therapy are attenuated for bystander apoptosis. Dr. Garg demonstrated in studies with Humanized mice and a fusion defective gp41 mutant that bystander apoptosis induction in the humanized mouse model is dependent on gp41 function. Dr. Garg also showed that the phenomenon of bystander apoptosis is dependent on CCR5 expression level as well as gp41 fusion. His studies provide the first experimental evidence for the reason why CCR5Δ32 individuals progress slowly to AIDS.
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Dr. Anjali Joshi
The Joshi lab is interested is studying the late stages of the virus life cycle mainly virus assembly and budding. Overall, the aim of her research is to provide novel insights into the molecular mechanisms governing retroviral replication and to apply this information towards the development of novel antiretroviral agents. The Joshi lab also aims to understand the basic biology of other important viral pathogens like Westnile virus and dengue virus aimed with the objective to not only advance the field of basic virus biology but also open novel avenues for treatment.
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David McClure
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Patent Protection

Provisional Patent Application Filed

Human immunodeficiency virus (HIV) over time causes acquired immunodeficiency syndrome (AIDS), a condition in which progressive failure of the immune system allows life-threatening, opportunistic infections and cancers to thrive.  Cell toxicity is a major problem when treating HIV.

This invention is a unique vector for modification of CD34+ hematopoietic stem cells for anti HIV gene therapy. The vector contains a modified version of the thymidine kinase gene, TKSR39, which can rapidly kill cells in the presence of gancyclovir (a drug already approved for Herpes virus treatment).

Features, Benefits & Advantages:

  1. The technology targets the infected cells and not the virus hence the development of resistance is unlikely.
  • By utilizing tat RNA to transiently express GFP in modified stem cells it’s providing for the first time a method to select modified stem cells that will increase the chances of a successful transplantation.
  • The vector is safe as it does not express the gene in the absence of HIV infection. Ganciclovir has been extensively used for herpes (CMV) treatment in HIV infected patients without significant toxicity or side effects.

Stage of Development:

Prototype is in testing