A fresh solution for gene therapy: nanostructures of polyvalent nucleic acids
Chemists at Northwestern University, working under the guidance of Professor Chad A. Mirkin, synthesized nanostructures from nucleic acids of a new type – polyvalent nucleic acids (Polyvalent Nucleic Acid Nanostructures, PNAN). These structures can be used to develop new methods of gene therapy for the prevention and treatment of various diseases.
The idea of gene therapy is to introduce DNA or RNA into a cell that can selectively "turn off" the genetic mechanisms associated with a particular disease. However, this approach is associated with a number of difficulties: it is necessary to deliver the therapeutic agent to the right cells, transport it inside the cells and avoid the development of undesirable side effects, which are often very serious. The use of new nanostructures makes it possible to solve most of these problems.
The T-alkyne molecules at the ends of the oligonucleotide chains attach to a catalyst–coated gold nanoparticle (NP - nanoparticle), which is then removed, leaving something similar to a knitted pompom or dandelion ball, consisting only of nucleic acids.
Due to the ability of nucleic acids to interact with proteins that provide endocytosis (the formation of membrane bubbles that capture particles located in all cells and their immersion in the cytoplasm), such nanospheres naturally quickly penetrate into the cell. This eliminates the need to use carriers causing undesirable side effects (positively charged polymers, peptides or viral vectors) used in traditional gene therapy methods to transport therapeutic DNA or RNA through the cell membrane.
According to Mirkin, the main advantage of the new nanostructures is that they open up huge prospects in the development of new methods of gene therapy for the treatment of a huge number of diseases, the development of which is based on known genetic mechanisms. Some of these diseases, for example, brain tumor glioblastoma, currently do not have effective therapeutic approaches.
The article by Joshua I. Cutler et al. "Polyvalent Nucleic Acid Nanostructures" was published on June 1 in the preliminary on-line version of the Journal of the American Chemical Society.
Evgeniya Ryabtseva
Portal "Eternal youth" www.vechnayamolodost.ru based on the materials of Northwestern University: A New Form of DNA.
08.06.2011