Nanomolecules Target RNA Diseases for Personalized Treatment

Using a chemical technique, researchers have found a way to not only identify and track cell damage, but to actually use those mutations to manufacture medication on site. This will allow healthcare professionals to more effectively diagnose and treat RNA-rooted disorders with lower costs and limited side-effect.

Personalized medicines made from nanomolecules are now at the forefront of a more efficient approach to fighting certain diseases.

Several genetic diseases stem from damaged RNA, but they are characterized by two copies of the faulty gene: one mutant copy responsible for causing the disease and one normal copy responsible for keeping the cell alive.

Creating more accurate drug delivery systems for diseases caused by damaged RNA depends on selective recognition of damaged versus undamaged alleles.

For instance, chemotherapy is one of the most effective treatments for cancer available, but can also paradoxically lower patient cure rates.

This is because the radiation employed during chemo targets cells indiscriminately, destroying both cancerous and healthy cells.

Therefore, it’s clear that the ability to selectively recognize the mutant copy is crucial to developing more targeted drugs with limited side effects, but was not possible until now.

Designer Drugs from Nanofactories

Researchers from the Scripps Research Institute, Florida campus have devised a way to first identify the damaged portions of RNA, and then use them to produce medication on-site.

Led by Matthew Disney, the team showed that it is possible to design nanomolecules that search, target and destroy only the RNA responsible for causing the disease. In other words, they’ve developed molecules tailored to selectively recognize the difference between larger alleles compromised by the disease and smaller alleles not affected by the disease.

The nano chemical technique developed by the team utilizes fluorescence lifetime imaging, or as Disney puts it, “we have brought RNAs out of the darkness and into the light by developing a chemical flare that goes off when a drug targets the RNA in a diseased cell and then continues to track the RNA’s movement.”

Beyond its search and destroy function, the team’s chemical technique can also transform disease-causing RNA into nanofactories that make their own personalized drugs.

More Effective Treatments Tailored to Your RNA Nanomolecules

Therefore, since fluorescence lifetime imaging offers a more efficient way to track RNA in diseased cells, the technique will allow more detailed study of how such mutations form. Coupling the increased tracking capability with the ability to deliver the perfect medicine to the perfect spot will lead to more effective treatments for RNA-associated disorders.

Coupling the increased tracking capability with the ability to deliver the perfect medicine to the perfect spot will lead to more effective treatments for RNA-associated disorders.

For example, the technique has the potential to cut costs and limit side effects with its ability to manufacture and deliver the right quantity of medication directly where it is needed and when it’s needed. The more tailored the treatment, the smaller the margin for error and complication.