Therapeutic Nucleic Acids: How Nanoparticles Can Be Engineered To Revolutionize Treatments

Genetic abnormalities, infectious illnesses, and cancer can all be effectively treated using nucleic acid treatments using DNA or RNA. However, research indicates that less than 1% of injected nucleic acid dosages reach target cells in an active state. Researchers underscored two of the main obstacles to this therapeutic approach with suggestions for overcoming the problems of therapeutic nucleic acids (TNA).

These are also highlighted in a study published in WIRES Nanomedicine and Nanobiotechnology.

According to Phys.org, the most difficult issues are delivered to the target cell and transported to the subcellular compartment where the nucleic acids are therapeutically active.

The authors examine how the composition of the nanoparticle can be engineered to manipulate or disrupt cellular membranes and facilitate delivery to the ideal sub-cellular compartments. They also look at how targeting molecules can be added to nanoparticle delivery systems to increase accumulation in particular cells.

"Controlling the sub-cellular delivery of DNA and RNA is the next major frontier for biotherapeutics. If we can overcome these barriers, DNA and RNA technology has the potential to revolutionize the treatment of a range of diseases," the corresponding author Angus P.R. Johnston, Ph.D., of Monash University, in Australia per Mirage News.

TNA's Problems

Dr. Changyi Johnny Chen, professor of surgery and molecular and cellular biology at Baylor College of Medicine said in a Medical Express report that a few TNAs have been cleared for use in clinical studies. He added that progress is hampered, mainly because TNAs are sometimes challenging to administer in living things.

There are various barriers that bare nucleic acids run across as they enter the body, which reduces their availability, researchers said. For instance, endothelial barriers may prevent them from achieving their optimal dispersion.

They may also face these challenges along with fast enzymatic digestion, liver retention, renal clearance, and unanticipated buildup in the "wrong" tissues. TNAs have serious negative effects and might trigger the immune system.

TNAs are promising methods to treat a range of illnesses despite the barriers restricting their applicability. The goal of Chen's lab is to create methods that, by enhancing their delivery, can raise their safety and effectiveness.

Liposomes, which are small, spherical lipid sacs that artificially develop around a water droplet carrying TNAs, are one of the most often employed delivery mechanisms. There have also been distribution methods based on viruses. Although both techniques have been helpful, there are issues with their efficacy and safety.

Solutions

The immune system makes proteins called antibodies that can identify and attach to particular cell surface signals, the same Medical Express report wrote. Targeting cancer cells while mostly sparing healthy cells, antibodies can mediate their delivery to particular cancer cells when they are coupled to other molecules or nanoparticles. These cancer cells express distinctive signals that are not present in healthy cells.

For instance, Chen and colleagues have previously demonstrated that some human malignancies, such as pancreatic cancer, have high protein mesothelin levels (MSLN) levels. MSLN, on the other hand, has little expression in the body's healthy tissues, making it a potential target for cancer therapy.

The enhanced nanoparticle delivery system is a technology platform that may be used to build novel medicines, molecular diagnostic techniques, or vaccinations for additional cancer kinds or disorders.

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