Development of Viral- and Non-viral-Based Hybrid Vectors

To date, viral and non-viral vectors have been widely used as two common strategies for delivering genes of interest to multiple target tissues, and they have been successfully used to treat a variety of genetic diseases such as cystic fibrosis, Leber's congenital amaurosis, and various severe combined immunodeficiency (SCID). Viral vectors are highly efficient at transducing genes but are immunogenic. Non-viral vectors have lower transfection efficiency, but they are generally less immunogenic. Both types of vectors have advantages and disadvantages that hinder their therapeutic endpoints in clinical trials.

To take advantage of the strengths of both types of vectors, researchers have attempted to develop hybrid vector combinations of viral and chemical vectors to achieve higher gene delivery efficiency than individual vectors alone. These hybrid vectors overcome the limitations associated with both delivery systems while enhancing desired features such as low immunogenicity, targeting ability, higher payload, and the ability to deliver multiple transgenes, thus making gene therapy a clinically available technology.

Category: General