DOVS News Research

Translating Science to Medicine | Tunable Gene Therapy

Shiming Chen, PhD, Dr. Bernard and Janet R. Becker Distinguished Professor in Ophthalmology and Visual Sciences

Gene therapy has been used in clinical trials to treat several types of inherited retinal degenerative diseases. Dr. Shiming Chen’s Lab aims to overcome the limitations of the current gene therapy technology by developing a new strategy called “tunable gene therapy” that has the ability to target diseases linked to mutations. 

The tunable gene therapy strategy allows the expression levels of a gene to be adjusted using a drug, thereby achieving optimal performance and low side effects. Dr. Chen is currently testing her new technology in animal models of retinopathies linked to mutations in the CRX gene – a gene that regulates the photoreceptor cells responsible for color and night vision. Too little or too much CRX can cause photoreceptor defects, resulting in blind spots. Thus, the expression of the therapeutic CRX gene needs to be carefully regulated, making it a good target for tunable gene therapy.

If Dr. Chen’s new tunable gene therapy strategy proves to be successful in animal models, it will open a door for new uses of gene therapy to treat diseases that are currently not under consideration. Dr. Chen is leading a human clinical study on CRX disease as a first step toward translating this new scientific advancement to a clinical therapy that may ultimately save the gift of sight for many patients. Dr. Chen has also applied for an international patent for her new technology with the hope that it can be used in treating retinal diseases as well as many other diseases.