Tailoring Treatment to Combat Diseased Cells at The Genetic Level


Source: University of Connecticut

Summary: Researchers have developed a new drug delivery system which uses a synthetic-biological hybrid nanocapsule for targeted treatment of many serious diseases at the genetic level.


With the regular standard therapies, delivery of a drug is rapid and total, and it is often insensitive to the levels of enzyme expression. They also increase the risk of over-medication, requires frequent dosing, and do not guarantee that the drug will reach the target cells. With these therapies, there are widespread adverse effects that can sometimes be worse than the illness being treated. Researchers from the University of Connecticut have developed a new drug delivery system which uses a synthetic-biological hybrid nanocapsule for targeted treatment of many serious diseases at the genetic level, at the same time leaves the healthy cells aside which increases the effectiveness of treatment and reduce unwanted side effects. The research findings were published in the journal Bioconjugate Chemistry.

synthetic-biological hybrid nanocapsule for targeted treatment

Enzyme-triggered degradation of a drug-loaded peptide-crosslinked nucleic acid nanocapsule. First a peptide is crosslinked at the nanoparticle surface (1), then an enzyme recognizes the peptide crosslinker (2), and finally the enzyme cleavage leads to the release of the drug and any intact DNA (3). Credit: Joseph Luciani/UConn

The delivery platform designed by the research team combines synthetic peptides, surfactants, and nucleic acids to form a nanocapsule which allows the time-appropriate, enzyme-specific co-release of a given pharmaceutical and an oligonucleotide (DNA or RNA). As a part of this study, they developed a unique linker technology to connect a synthetic drug delivery vehicle called as a nucleic acid nanocapsule (NAN) with a new peptide cross-linker approach. This combination produces a nanocapsule capable of guiding genetic or pharmaceutical molecules to a target on or within a cell. Prof. Rogue feels that this method has a clear promise to reduce the negative side effects in cancer patients undergoing chemotherapy and could be applied to many genetic and acquired diseases.

Asst. Prof. Jessica Rogue, “There’s no one-size-fits-all delivery system”, “The beauty of this system is that it is programmable, modular, and has the ability to rapidly integrate diverse peptide sequences. It can be tailored to combat new disease challenges as they emerge.”


More Information: Joshua J. Santiana et al. Programmable Peptide-Cross-Linked Nucleic Acid Nanocapsules as a Modular Platform for Enzyme Specific Cargo Release, Bioconjugate Chemistry (2017). DOI:10.1021/acs.bioconjchem.7b00629


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