A novel switch to turn genes on/off on cue, a promising step toward safer gene therapy

Just like a doctor adjusts the dose of a medication to the patient's needs, the expression of therapeutic genes, those modified in a person to treat or cure a disease via gene therapy, also needs to be maintained within a therapeutic window. Staying within the therapeutic window is important as too much of the protein could be toxic, and too little could result in a small or no therapeutic effect. Researchers now report on a technology to effectively regulate gene expression, a promising solution to fill this gap in gene therapy clinical applications.

Just like a doctor adjusts the dose of a medication to the patient's needs, the expression of therapeutic genes, those modified in a person to treat or cure a disease via gene therapy, also needs to be maintained within a therapeutic window. Staying within the therapeutic window is important as too much of the protein could be toxic, and too little could result in a small or no therapeutic effect. Researchers now report on a technology to effectively regulate gene expression, a promising solution to fill this gap in gene therapy clinical applications.

Read more https://www.sciencedaily.com/releases/2024/01/240102142035.htm