New research could lead to new treatments for genetic diseases

(ABC 6 News) – The Hormel Institute, University of Minnesota’s, George Aslanidi, PhD, has published research that could lead to improved access to personalized treatment for patients with genetic diseases.

With more than 7,000 genetic diseases with limited treatments available, the need for personalized approaches to disease treatment is ever present.

In the past few years, the U.S. Food and Drug Administration has approved several genetic drugs based on the Adeno-associated virus (AAV). AAV is a virus that can be designed to deliver DNA to targeted cells. However, the lack of a personalized approach to find the most appropriate type of AAV for treatment means these treatments aren’t effective for all people who need it.

Dr. Aslanidi and his team have developed and validated a novel laboratory test used to find and measure the amount of a specific substance that can help to identify which AAVs may best meet eligibility requirements for patients with genetic diseases to enroll in appropriate clinical trials.

“Our test has an advantage over currently used methods because it measures the effectiveness of multiple genetic drugs on a small volume of patients’ blood under the identical conditions,” said Dr. Aslanidi. “This increases the accuracy and reproducibility of the results and could lead to further development of the treatment options for patients with genetic diseases, and possibly cancer.”

Dr. Aslanidi plans to conduct further studies to continue to validate the test and work with
clinical doctors to match patients’ information with particular drugs formulation needed for
treatment.

The article “Multiplexing AAV Serotype-Specific Neutralizing Antibodies in Preclinical Animal
Models and Humans” was published in the journal Biomedicines. The full article is available
here.