Gene therapy represents an innovative approach to treating a wide range of diseases, including cancer, diabetes, and inherited metabolic disorders. Despite promising results, one of the main challenges limiting its practical application is the delivery of genetic material to target cells in the body. The dosage of genetic material and its effective introduction into cells often lead to high costs and risks associated with the immune system.
A new study by researchers from the University of Wisconsin-Madison, published in PLOS ONE, demonstrates a promising solution to this problem by utilizing electric pulses that make human cells more susceptible to absorbing genetic material. This discovery could open new avenues for more accessible and safer gene therapy.
Direct delivery of genetic material to target cells offers several significant advantages over systemic methods, where material is introduced into the peripheral bloodstream. One key advantage is that it reduces the overall dosage of genetic material required. In systemic delivery, a large portion of the genetic material is lost during its passage through the bloodstream and organs, necessitating significantly larger doses. This not only increases production costs but also raises the risk of adverse reactions, such as excessive immune response or cytokine storms. A cytokine storm is a severe immune reaction in which the body produces excessive amounts of inflammatory molecules, potentially leading to tissue damage.
By utilizing direct delivery, the time genetic material spends in circulation is reduced, thereby minimizing the likelihood of immune response and loss of effectiveness. This significantly increases the safety and accessibility of the therapy for more patients.
The researchers at the University of Wisconsin-Madison have developed a technique where electric pulses are used to enhance the absorption of genetic material by cells. Although the exact mechanism by which electric pulses increase absorption is not fully understood, studies indicate that the pulses create small (nano) pores in the cell membrane, facilitating the entry of genetic material into cells.
One of the technologies employed involves viral vectors, such as AAV8 (adeno-associated viruses), which are effective carriers of genetic material. Despite the relatively large size of these vectors, scientists believe that creating pores in the membrane may ease their penetration. However, this remains a subject for future research.
Effective delivery of genetic material could make gene therapy more accessible and safer for millions of people. Many inherited metabolic diseases, such as diabetes, hemophilia, and cystic fibrosis, affect the liver, which plays a central role in metabolism. Advanced methods for delivering genetic material to liver cells could correct genetic mutations and offer lasting treatment for these conditions.
The development of new methods for delivering genetic material to target cells could significantly reduce treatment costs. Implementing techniques like electric pulses could make these therapies available to a broader range of patients.
The next phase of research involves optimizing the parameters of electric pulses, such as intensity, duration, and number of pulses, to maximize the effect on the absorption of genetic material without damaging cells. Additionally, the scientists plan to transition from in vitro experiments on cell cultures to in vivo studies in laboratory conditions. Long-term plans include expanding the application of the technique to other cell types and organs, aiming to broaden treatment possibilities for various diseases.