Research Team Develops Technology That Could Make Gene Therapy Safer

A Baylor College team of researchers headed by author and Associate Professor of Pathology, Liaising Yen, have spent over ten years working on a technology to effectively regulate gene expression.

As outlined in a recent article in Science Daily and published in Nature Biotechnology, Prof. Yen’s technology is considered to be a solution that satisfies the gap in clinical applications for gene therapy.

About Gene Expression

The majority of genes possess all the information required to produce proteins (molecules). Moving from gene to protein is a complex process consisting of two steps: transcription and translation, collectively known as gene expression.

It is an on/off switch which controls where and when RNA proteins and molecules can be made. The new technology effectively regulates gene expression.

Beginning with the process of transcription, the information that is stored in the DNA of a gene is then passed along to the RNA molecule in the cell nucleus.

Genes may be modified by gene therapy to treat a disease after which they must take one more step. The genes must be maintained within a therapeutic window.
The therapeutic window (or pharmaceutical window) of a drug is the range of drug dosages which can treat disease effectively without having toxic effects.

Its principle has been recognized for quite some time. However, until now there were no strategies for safe implementation. Therefore, potential applications of gene therapy have been limited.

Think in terms of a doctor prescribing medication and adjusting the medication in accordance with a patient’s needs. Accordingly, using genes that are modified in order to treat a disease must be accomplished in the therapeutic window.

The importance of staying within a safe range is that too much protein may be toxic and administering too little protein may leave the patient with no therapeutic benefit.

The professor told Science Daily that their technology uses small molecules that interact with RNA and may not cause an immune response. The system developed by Prof. Yen and colleagues turns genes on at various levels with the use of small molecules that are doses approved by the FDA.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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