If You Know the Genes to Cure a Disease, We Show How to Deliver Them

Published on : Dec 21, 2019

Nature can probably be an unlikely guide to scientists to test new approaches in gene therapy. Researchers have hit some success in finding effective gene combinations for diseases, notably cancer. World over, they have come out with good number of gene combinations to this end. But most importantly, offloading the cargo correctly and at the right amount is nothing short of challenge.

Nanocarrier Exosome Could Pass Blood-Brain Barrier

In a proof of concept, a team of researchers have demonstrated a new gene-therapy technique using a promising biologically friendly carrier-exosome. The method harnessed human cells to produce tiny nano-sized particles at scale, which can deliver drugs with therapeutic potential. Interestingly, the regenerative materials that the nanoparticles exosomes carried could even pass blood-brain barrier. Furthermore, they showed that the method is effective to reverse disease processes in mice models with gliomas. These are cancer that account for overwhelming percent of malignant brain tumors in humans. The researchers considered this process as a novel approach-the one waiting to form part of gene therapies for numerous diseases. These include Alzheimer's and Parkinson's.

The technique comprised generating exosomes from tens of thousands of donated cells-one million to be precise-notably consisting mesenchymal cells collected from human fat. In addition, using electrical stimulation and a process they call DNA force-feeding, the investigators could produce large numbers of exosomes. Further, they used the nanoparticles to transport a gene called PTEN. The mutation in PTEN causes proliferation of the cancer cells.

Targeted Delivery of Large Genetic Instructions to Reverse Disease Processes

Exosomes are tiny, but large numbers of them can pack sufficient genetic information to show therapeutic effect in cancer. Two prominent advantages that the method offers are that exosomes are bereft of any toxicity and can permeate blood-brain barrier. Stridently, they not just found the cancer cells but also killed them, asserted the researchers.

They used a patented technology one of the researchers had found in 2017 called tissue nanotransfection (TNT). The journal Nature Biomedical Engineering published the study.