Available Technologies

Technology

Scientists have been working on gene therapy for nearly 30 years. Gene therapy involves delivering a nucleic acid into the patient’s cells to treat a disease. Currently there are no effective ways to introduce the nucleic acids into the cells without a carrier, also known as a vector. There are two vectors that are commonly used, recombinant viruses and non-viral vector methods such as electroporation. Recombinant viruses have several critical drawbacks, such as toxicity, while non-viral vectors are known to be inefficient.

Researchers at UAH have developed a new carrier method known as, Cytokine-Receptor Mediated Delivery of Nucleic Acids. This new method provides a non-toxic, non-viral, and efficient carrier of nucleic acids utilizing a  PEI-Cytokine conjugate.

The PEI-Cytokine technology developed at UAH provides a non-toxic and efficient vector. Over the past ten years, there has been a growth in the amount of gene therapy research and testing. There have been several cases of successful gene therapy routines curing rare diseases, sickle cell disease, and even cancer. The tremendous progresses recently made in gene editing technologies (e.g. CRISPR-Cas9) and adoptive cell therapies using genetically modified cells (e.g. CAR-T) significantly increase demand for a safe and efficient nucleic-acid delivery technology. This could expedite research and testing in the gene therapy field. The PEI-Cytokine vector technology meets such imminent and critical needs, providing the platform for a multitude of disease cures to be developed.