Boris Kantor, PhD, Duke University School of Medicine, Durham, NC, highlights ongoing efforts to develop novel techniques and technologies for gene therapy in neurodegenerative diseases, in order to maximize safety and efficiency. Dr Kantor highlights his ongoing work using integration-deficient lentiviral vectors to create a system that benefits from robust delivery, efficient transduction, and large cargo capacity, while avoiding the risks of insertional mutagenesis. Researchers are also looking into innovative ways to use small molecules to enhance the precision and safety of genome editing systems like CRISPR/Cas. Small-molecule modulation of Cas nuclease and gRNA expression levels, small molecule SpCas9 inhibitors, and targeted degradation/stabilization of Cas nucleases by small molecules are all being studied. Screening for smaller Cas9 variants is another focus, to overcome the need for dual-vector delivery which introduces significant caveats. This interview took place at the Alzheimer’s Association International Conference (AAIC) 2022 in San Diego, CA.
