Study highlights need for standardized measurement methods in gene therapy

Medical Xpress January 6, 2025
Lawrence Goodman, National Institute of Standards and Technology

In a finding with implications for one of the most promising cutting-edge medical treatments, researchers evaluated several measurement techniques commonly used in gene therapy. The study determined that one of the most popular techniques was “problematic” and requires further development and standardization.

In gene therapy, a person’s faulty genes are either replaced or modified to treat or prevent disease. With roughly two dozen products on the market and hundreds of clinical trials in progress or planned, the treatment is hailed as a revolutionary method for targeting the root genetic causes of diseases ranging from sickle cell to cancer.

Some gene therapy treatments use modified adeno-associated viruses (AAVs) to deliver therapeutic genetic material into a patient’s cells. These AAVs are engineered...