Some gene therapy treatments use modified adeno-associated viruses (AAVs) like the one shown here to deliver therapeutic genetic material into a patient’s cells.
Credit: Thomas Cleveland (IBBR/NIST)
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 to target specific cell types. Then their infectious genetic material is replaced with the therapeutic genetic material, and they are administered to the patient.
Correctly measuring AAV vectors, as they are called, is critical to their safety and efficacy.
In a recently released study, researchers at the National Institute of Standards and Technology, the National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), and the U.S. Pharmacopeia (USP) recruited six industry labs in the United States and Europe to measure sample AAV vectors.
The labs were asked to quantify ..
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