脑损伤
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Figure 1｜Schematic showing future prospects for the development of brain-targeted gene delivery vectors.
With the rapid expansion of the concept of pharmaceutics, including regenerative medicine such as induced pluripotent stem cells and 3D printer drug discovery, gene therapy is expected to become the next innovative medicine after small molecular drugs and antibody drugs. Gene therapy is thought to be particularly promising for treating CNS disorders including Alzheimer’s disease, one of the most socially burdensome diseases. A non-invasive targeted vector is attractive and would expand the range of gene therapy applications. There is no doubt that research focusing on both viral vectors and non-viral vectors for gene therapy will be further accelerated in the future (Figure 1). Although high-throughput screening systems such as using the DNA barcoding method are available, it will be necessary to investigate the interaction mode and response between the vectors and the body in more detail. In addition, quantitative data regarding the amount of therapeutic gene expression that are needed to cure the diseases, which is important for determining the dose needed for gene therapy and for achieving both safety and efficacy will be needed. Gene delivery research has made dramatic progress in the past decade, but there is still room for improvement, especially in delivering cargoes to the brain. Further development is still needed and desired.