Seigo Kimura

Hokkaido University, Japan

Title: Overview of CNS-Targeted Gene Delivery across the BBB: Non-Invasive Delivery Strategies

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Biography

Mr. Seigo Kimura is a first year doctoral student in the field of drug delivery especially in gene delivery at Hokkaido University studying under Professor Hideyoshi Harashima. The aim of his studies is to develop safe and efficient drug delivery system for nucleic acid medicine and gene therapy. He focuses on lipid-based carriers to address some of issues that exist in the process of nucleic acid delivery in vivo. More specifically, he has been working on the development of non-viral vectors using plasmid DNA-encapsulated lipid nanoparticles (LNPs). His previous studies regarding LNPs capable of in vivo gene transfer to immune cells and those applicability to DNA vaccines have been published in J Control Release. Currently, he has begun work on gene delivery to the brain and is working to develop LNPs for the delivery of therapeutic genes to the brain through a non-invasive delivery route. 

Abstract

The era of the aging society has arrived, and this has been accompanied by an increase in the absolute numbers of patients with neurological disorders, such as Alzheimer’s disease and Parkinson’s disease. The most important point in the current situation is that there is no effective treatment despite the fact that the number of patients increase with the aging of the population. The bottleneck in drug development for CNS diseases is the absence of effective drug delivery system (DDS) technology for delivering the therapeutic agents into the brain. While gene therapy has great promise for the treatment of neurological disorders, gene therapy is the field where DDS technology is most needed due to low membrane permeability and low stability of nucleic acids in vivo. Thus, the development of brain-targeted DDS is as important as or even important than drug itself. Nanotechnologies such as viral and non-viral vectors allow efficient brain-targeted gene delivery systems to be created. In 2019, the FDA approved a gene therapy for spinal muscular atrophy (SMA), Zolgensma. The advent of Zolgensma confirmed that in vivo targeted gene therapy is a clear possibility and is expected to further accelerate the development of DDS technology in anticipation of gene therapy. The topic of this session is an overview of CNS-targeted gene delivery across the blood brain barrier (BBB) via non-invasive delivery strategies based on currently published my review article entitled with “Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB”. This session mainly address two aspects of this situation: (1) BBB receptors/transporters in terms of BBB crossing; (2) non-invasive brain-targeted strategies mainly by non-viral methods. Although the present state of CNS-targeted drug development is still in the initial stage, the purpose of this session is to provide current situations and to inspire future persistent researches.