The new strategy for co-administration of therapeutic RNA and versatile drugs can be used to effectively treat cancer, viral infections and other complex diseases.
Getting past the Endosomal Barrier
Since its discovery, one of the largest obstacles in siRNA therapy has been delivering these molecules to where they need to be with out losing their ability. The researchers instead found a creative solution to this.
By attaching siRNA to ionizable drugs, the stability and cellular delivery efficiency of siRNA can be greatly improved. The siRNA and drug must be released into the cytoplasm in order to function, thus using ionizable drugs, which change their charge based on pH levels, allowing them to help the two escape from the endosome.
Different from most existing delivery methods that encapsulate siRNA and drug separately in a single nanoparticle, the novel method uses lipid for the natural protective effect on the siRNA while delivering each RNA and drug together into the inside of target cells. The discovery is an advance on the endosome barrier — a major problem in siRNA therapy that has been around for nearly two decades.
Dual Delivery for Multiple Conditions
In addition, their co-formulation method enables siRNA to enter the cells along with an active a weak ionizable drug at same time. This could represent a breakthrough in the treatment of diseases characterised by the involvement of multiple pathways, specifically cancer and viral infections.
Now siRNA is known as the “magic bullet” and is a potent weapon against gene silencing that causes diseases. But until now, conventional nanoparticle formulations were not capable enough to pack in a large number of small molecule drugs. This drawback is what the team has overcome with their discovery of using ionizable drugs as carriers that can cotransport siRNA and drug effectively.
Such new method opens the door for successful strategies in diseases that need more than one punctual approach. The method combines, for the first time, the silencing power of siRNA with the therapeutic action of drugs to address multiple aspects and multiple challenges that have complicated treatments for many conditions.
Conclusion
The scientists hope that their delivery of therapeutic RNA and powerful drugs straight into cells can lead to a plethora of new treatments being developed in the future. This new strategy would be highly significant in overcoming one obstacle — the endosome barrier, and allowing simultaneous delivery of siRNA and drugs to treat complex diseases such as cancer and viral infections. In this meticulously calculated procedure and the effects it can have in practice, is an excitement that has been generating ripples throughout the scientific community — alluding to a future of personalized medicine like we have never seen before.
