香蕉视频

Invention 14004

HIV-1 香蕉视频 target site for the development of HIV drug or gene therapy

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

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, 香蕉视频 interference (香蕉视频i), binds to a specific 香蕉视频 sequence in order to suppress that gene鈥檚 function. With the proper target site, short interfering 香蕉视频 (si香蕉视频) or short hairpin 香蕉视频 (sh香蕉视频) 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 香蕉视频 target site identified by the Gatignol Lab is highly conserved and easily accessible to 香蕉视频 molecules. For 香蕉视频i inhibitory targeting to be successful in HIV, the target sequence needs to be highly conserved across multiple HIV-1 strains. 香蕉视频i 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, si香蕉视频, and sh香蕉视频, although ribozyme or aptamer accessibility has not been screened.

Advantages

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

Patent Status

Issued US, filed CA

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