Many diseases have at their origin a protein that does not function properly. Now, a multidisciplinary research team with Texas A&M AgriLife and Texas A&M University has found a way to deliver a protein quickly, effectively and briefly to the brain, with therapeutic and scientific implications.

Graphic shows a neuron with protein and delivery tool molecules coming toward it
The new delivery tool (key symbol) and a target protein (shown in green) are mixed in solution and injected into the target site. The delivery peptide enables the protein to enter a cell’s endocytic pathway, then the cytoplasm and nucleus. (Graphic courtesy of Jean-Philippe Pellois.)

Potential uses for the method in the future could include repairing spinal cord injuries and a range of other localized injection applications.

“We found that we could successfully deliver a protein into mouse brains,” said Jean-Philippe Pellois, Ph.D., professor and associate head for graduate program, Department of Biochemistry and Biophysics in the Texas A&M College of Agriculture and Life Sciences. “Proteins are large molecules that don’t easily enter cells or cross cell membranes, but we’ve created a trick to achieve this.”

Both the protein and its delivery system degrade naturally after performing their role.

“We wanted to make sure we had reagents that are very gentle on the cell, that can enter cells without disrupting them and then leave without a trace,” said Pellois, who is also a researcher with Texas A&M AgriLife Research.

Pellois and his lab collaborated with the lab of Cédric Geoffroy, Ph.D., assistant professor in the Department of Neuroscience and Experimental Therapeutics in the Texas A&M School of Medicine.

Funders of the study included the National Institute of General Medical Sciences, the Cancer Prevention and Research Institute of Texas, the Craig H. Neilsen Foundation, and The Institute for Rehabilitation and Research Foundation.

The results, “In vivo peptide-based delivery of a gene modifying enzyme into cells of the central nervous system,” appeared Sept. 28 in the peer-reviewed journal Science Advances.

Read more on AgriLife Today.