Genome Surgery

Description:

Invention Summary

The invention is a development in a technology to delete large regions of genomic DNA in people, animals or bacteria. It uses a three-component system consisting of a cell permeation component, a sequence specific DNA binding component, and an endonuclease component. Further, the invention can remove active and latent viral genomes. Delivery can be by injection of the purified three-component protein.

Market Opportunity

The invention can be applied for treatment of many diseases—both of infectious and non-infectious nature. Currently, there is no way to delete pieces of DNA unless cells are removed from the body, manipulated, and implanted back in the body. Also, in gene therapy, there is no way to remove bad copies of genes. The invention fills these voids.

Features & Benefits

A means for excising the HIV genome from humans, helping to reduce or eliminate HIV infection including latency.

Removal of DNA-based genomes of viruses from host cells. Bacterial infections can also be targeted.

Target cancer. One approach would be to target proto-oncogenes and oncogenes by local introduction of the construct into the local region of the tumor. Another approach would be disabling endogenous apoptosis inhibitors such as BAD and Bcl2 in host cells with the goal of encouraging apoptosis of cancer cells.

Fight non-infectious diseases. In many diseases, patients have genes with bad alleles, which cause proteins to misfold resulting in pathologies. Examples are Lewy bodies in Parkinson’s disease, amyloid plaques in Alzheimer’s disease, and protein insolubility in triplet repeat diseases such as in Huntington’s disease.

Treat autoimmune diseases. Remove specific harmful antibodies that generate immune responses in the 100’s of autoimmune diseases such as type 2 Diabetes and Lupus.