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.

Although the principle of therapeutic window has been known for a long time, there has been no strategy to implement it safely, limiting the potential applications of gene therapy in the clinic.

In their current study published in the journal Nature Biotechnology, researchers at Baylor College of Medicine report on a technology to effectively regulate gene expression, a promising solution to fill this gap in gene therapy clinical applications. A Research Briefing on the breakthrough has been published in the same journal issue.

Unlike older methods that use things foreign to our bodies, this one doesn’t trigger our immune system and employs small molecules to interact with RNA.

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Researchers at Baylor College of Medicine have developed a breakthrough technology to regulate gene expression in gene therapy, addressing the crucial issue of maintaining therapeutic gene levels within a safe range.

This is important because having too much or too little of a gene’s activity within a therapeutic window can cause problems. Their method uses tiny substances in amounts approved by the FDA to control the genes.

Current methods have issues

The current methods for regulating genes have some problems, like causing harmful immune responses.

From mRNA technology to T cell engagers, there were a number of advancements in cancer research in 2023.

Scientists introduce Zman-seq, a method revolutionizing our understanding of dynamic cellular changes in the human body over time. Read more about this groundbreaking study.

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In a recent study published in Cell, scientists led by Prof. Ido Amit at the Weizmann Institute of Science have introduced Zman-seq. This revolutionary method breaks through the temporal barriers of cellular analysis.

This innovative approach allows tracking and measuring changes in individual cells within the body over time.

“Knowing what preceded what is not enough to deduce causality, but without this knowledge, we don’t have a chance of understanding what the cause and effect are,” said Prof. Amit in a press release.