New Study Shows Promise for Genetic Medicines as a Systemic Therapy

Introduction

The use of genetic medicines to produce proteins to replace missing or defective ones has been studied and used for many years. However, these therapies typically keep the produced proteins inside cells. Researchers at the UT Southwestern Medical Center have found a way to guide proteins to where they are needed, offering potential for systemic therapy that could improve the effectiveness of current protein drugs and enhance the health and quality of life for patients with a wide range of conditions. This article will explore the details of the new study, its findings, and the potential implications for the field of healthcare.

What is the Study About?

In a new study, researchers at UT Southwestern have found that delivering genetic material with a cellular “ZIP code” prompted cells in mouse models to produce and release proteins or drugs into the bloodstream. The researchers used signal peptides (SPs) as cellular “ZIP codes” to guide proteins where needed. They focused on secretory SPs, which direct proteins to be released from cells into the bloodstream. The researchers engineered mRNA fragments encoding a secretory SP from Factor VII, a protein in blood clotting. They attached them to mRNA for proteins like mCherry (for visualization), erythropoietin (blood production), etanercept (anti-inflammatory), and anti-PD-L1 (cancer treatment). These modified mRNAs were then packaged into lipid nanoparticles and induced cells in lab dishes to secrete the proteins into the surrounding fluid, demonstrating a potential new approach for systemic therapy.

What Are the Potential Implications?

The researchers were inspired to explore whether the body could produce and release therapeutic proteins like a biofactory. They tested this by treating mice with psoriasis using modified mRNA for etanercept, which significantly reduced skin plaques and inflammation markers. Similarly, treating mice with two types of cancer using modified mRNAs for anti-PD-L1 slowed tumor growth and doubled survival compared to untreated mice.

Advantages Over Intravenous Treatments

The findings of the study suggest future therapies where patients’ bodies make their medicines, potentially offering advantages over intravenous treatments. Daniel Siegwart, Ph. D., Professor of Biomedical Engineering and Biochemistry at UT Southwestern, highlighted the potential for patients to receive treatments less frequently, improving their quality of life. This technology also simplifies treatment schedules, reducing the need for frequent and lengthy infusions. Using the body’s processes to make and deliver therapeutic proteins could reduce side effects and improve the effectiveness of current protein drugs that require infusion.

What Are the Potential Applications?

This technology has the potential to enhance health and quality of life for patients with a wide range of conditions such as inflammatory diseases, cancers, clotting disorders, diabetes, and various genetic disorders. As the technology progresses, it can potentially lead to systemic therapy that can target a range of diseases with high efficacy and precision. The technology is still in its infancy, and more studies need to be conducted to optimize its use for various types of diseases.

Conclusion

The use of genetic medicines to produce therapeutic proteins for systemic therapy is a promising field of research. The findings from the new study by UT Southwestern Medical Center show a potential new approach that uses the body’s processes to deliver therapeutic proteins. This could potentially lead to therapies that are less invasive, more effective, and have fewer side effects. As the technology develops, it could provide an effective alternative to traditional intravenous treatments for various diseases.

Originally Post From https://www.techexplorist.com/bioengineering-body-self-produced-medicine/86052/

Bioengineering the body for self-produced medicine