New RNA-based tool can illuminate brain circuits, edit specific cells: Editing technology is precise and broadly applicable to all tissues and species

Duke University researchers have developed an RNA-based editing tool that targets individual cells, rather than genes. It is capable of precisely targeting any type of cell and selectively adding any protein of interest.

Researchers said the tool could enable modifying very specific cells and cell functions to manage disease.

Using an RNA-based probe, a team led by neurobiologist Z. Josh Huang, Ph.D. and postdoctoral researcher Yongjun Qian, Ph.D. demonstrated they can introduce into cells fluorescent tags to label specific types of brain tissue; a light-sensitive on/off switch to silence or activate neurons of their choosing; and even a self-destruct enzyme to precisely expunge some cells but not others. The work appears Oct. 5 in Nature.

Their selective cell monitoring and control system relies on the ADAR enzyme, which is found in every animal’s cells. While these are early days for CellREADR (Cell access through RNA sensing by Endogenous ADAR), the possible applications appear to be endless, Huang said, as is its potential to work across the animal kingdom.

“We’re excited because this provides a simplified, scalable and generalizable technology to monitor and manipulate all cell types in any animal,” Huang said. “We could actually modify specific types of cell function to manage diseases, regardless of their initial genetic predisposition,” he said. “That’s not possible with current therapies or medicine.”

CellREADR is a customizable string of RNA made up of three main sections: a sensor, a stop sign, and a set of blueprints.

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