Diseases like cystic fibrosis, hemophilia, Duchene’s muscular dystrophy, and Huntington’s disease are single genetic mutations which cause serious, life-threatening problems. The only permanent treatment for them is to replace the defective gene. The issues involved in curing genetic disease this way are reoccurring problems with:
- immune tolerance to the new/functional gene
- getting the gene to the right place in the body
- stably expressing the gene for the lifetime of the individual
Our smart drug delivery technology helps to overcome these challenges in treating genetic diseases. If you are interested in learning more, please contact us.