In this special eBook we explore the challenges and opportunities in bioanalytical support for gene therapy medicinal product development and the techniques used to develop gene therapies.
Thousands of people are born with diseases caused by genetic defects every year. A gene mutation can result in the absence or reduced function of a critical protein. Gene therapy, a recent and fast-growing area of medicine, promises to change the way we approach and treat these diseases. Traditionally a major hurdle has been delivering an effective gene copy to cells, with early viral-based methods proving highly immunogenic. With an excellent safety profile and the ability to thrive in a range of human tissues, the adeno-associated virus (AAV) has reinvigorated the field of gene therapy.
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