Nanosyringe Shows Promising Results for Gene Therapy, Cancer Treatment [Study]

A novel injection system has been discovered, and it can revolutionize gene therapy. The nanosyringe can be used to tweak genes.

Nanosyringe for Gene Therapy Delivery

Researchers report modifying Photorhabdus's syringe-known as a contractile injection system, so it may connect to human cells and inject big proteins into them.

The research may offer a method for delivering diverse therapeutic proteins-including those that can "edit" a cell's DNA - into any cell. Mark Kay, a Stanford University gene therapy expert uninvolved in work, said it was a pretty interesting approach. He was convinced that the strategy would be valuable when one wants to create proteins that can do genome editing to fix or knock out a gene that is mutated in a genetic condition, Scientific American reported.

The nano injector might be a vital tool for researchers interested in tinkering with genes. According to study investigator Feng Zhang, a molecular biologist at the McGovern Institute for Brain Research at the Massachusetts Institute of Technology and the Broad Institute of M.I.T. and Harvard University, delivery is the most challenging part of gene editing. Zhang is renowned for creating the CRISPR-Cas9 gene editing method.

The editing apparatus can currently be inserted into a few tissues, blood, the liver, and the eye. However, they didn't have an excellent way to get it anywhere else, including the brain, heart, lungs, or kidneys. The new nanosyringe can bind to specific tumor receptors making it a promising cancer treatment.

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When Zhang and his doctoral student Joseph Kreitz read two publications on Photorhabdus's injection technique two years ago, they explored novel approaches to deliver gene-editing enzymes to cells. Because it was tailored to insect cells, the system was special. According to Zhang, this is one of the extremely few instances where a bacterial entity enters an animal cell as opposed to another bacterial cell. They hoped that if something could be injected into animal cells, it could also be effective on human cells.

Nanosyringe Is Specific, Can Pack System With Protein Payloads

The team successfully got the altered tail fiber nanosyringes to adhere to an epidermal growth factor receptor (EGFR) found on the surface of some human cancer cells in one experiment. A poison loaded into the injection system killed almost all the cells containing the receptor but did not affect other cells, demonstrating its selectivity. The researchers modified the injectors' tail fibers to recognize surface markers on many different cell types.

Zhang's team also discovered that by labeling the proteins with a tag that designates them as ammunition that must be loaded onto a syringe's needle, the system might be loaded with various protein payloads.

The gene-editing enzyme Cas9, a sizable molecular scissor that snips DNA at a position defined by a molecule, guides the scissors to the proper place. The researchers gave protein toxins this moniker.

The proteins either destroyed the cells or altered their genes when introduced to human cells. According to Zhang, the study demonstrated that loading several protein types onto these needles simply by attaching a tag to the protein is possible. Additionally, each needle can also load multiple copies of a protein to improve the dosage.

The study is published in Nature.