Advanced Cell and Gene Therapy

Drug Delivery Strategies for Gene Therapies

Drug delivery strategies for gene therapies are critical for ensuring that therapeutic genes are effectively delivered to the target cells and tissues. These strategies involve the use of various delivery vectors and techniques to enhance the efficiency, specificity, and safety of gene therapy. AAVs are widely used due to their low immunogenicity and ability to deliver genes to both dividing and non-dividing cells. They are suitable for long-term gene expression and have been used in treating genetic disorders like hemophilia and retinal diseases. Lentiviral vectors can integrate into the host genome, allowing for stable and long-term expression of therapeutic genes. They are commonly used in ex vivo gene therapies, such as CAR-T cell therapy for cancer. Adenoviral vectors can deliver large genetic payloads and achieve high levels of gene expression. However, they can provoke strong immune responses, making them more suitable for short-term therapies