AAIC 2022 | Viral-mediated gene therapy for neurodegenerative disease

Boris Kantor, PhD, Duke University School of Medicine, Durham, NC, discusses the pros and cons of current virus-mediated tools for gene transfer and the technologies under investigation to overcome their shortcomings. While gene therapy is a highly promising approach for neurodegenerative disease, work is needed to improve upon current delivery systems. Viral vectors derived from human immunodeficiency virus type 1 (lentiviral vectors) have a large capacity and efficient packaging and transduction capabilities but are limited by their integrative nature. Integration-deficient lentiviral vectors are under investigation to create a system that benefits from robust delivery and large cargo capacity, while avoiding the risks of integration. Adeno-associated virus (AAV) vectors are very commonly used given their safety, but they have a small packaging capacity, making them difficult to use for bulky cargo like an all-in-one CRISPR/Cas system. Other delivery methods are also under investigation, such as nanoparticles and microparticles. This interview took place at the Alzheimer’s Association International Conference (AAIC) 2022 in San Diego, CA.