Advanced (Gene and Cell) Therapies for Central Nervous System Applications

Gene therapy comprises of a set of strategies to modify gene expression or correct mutant genes. Gene augmentation through the introduction of a functional gene, gene silencing by RNA interference, and gene editing with CRISPR technology have become powerful and instrumental approaches in the development of new medicines. Several gene therapy strategies using viral and non-viral vectors have been developed and tested with success for CNS application. Nevertheless, improved vector development and complete safety elucidation of these strategies is still required. On the other hand, cell therapy relies on the use of cells and tissues to repair, regenerate, or replace affected cells and tissues resulting in a restoration of the organs´ normal function. Several cell types, namely, adult, fetal, and embryonic stem cells, and induced pluripotent stem cells, can be used in transplantation to promote cell replacement and neuroprotection. Still, several key aspects concerning cells´ safety and therapeutic potency remain to be better understood.



This Research Topic focuses on Advanced Therapies for CNS application and will provide a broad overview of the latest developments in the Gene and Cell Therapy field. It will cover some of the critical aspects for research and clinical implementation of gene- and cell-based therapies applied to the CNS. Moreover, it will discuss new tools and techniques for gene and cell therapy medicine development. A particular emphasis on the development and preclinical and clinical testing of therapeutic targets, viral and non-viral vectors for gene therapy, and cell sources for cell therapy will be presented. Key features of the gene therapy vectors development, such as gene therapy tools delivery efficiency, off-target effects and immunogenicity, and cell and CNS targeting, will be considered. Key features of cell therapy products, such as different cell types available for brain application, cell isolation and reprogramming strategies, cell characterization for identity, potency and purity, therapeutic effects after CNS transplantation, tumorigenicity, immunogenicity, and grafts overgrowth, will also be taken into consideration.



We welcome Original Research, Review, Mini-Review, Clinical Trial, Case Report and Opinion articles that cover, but are not limited to, the following topics:



• The latest advances in gene- and cell-based therapies (including medicines) applied to the CNS;

• Gene Therapy strategies promoting gene augmentation, silencing, and editing in the CNS and CNS experimental models, such as brain organoids;

• Development, preclinical and clinical testing of gene editing, augmentation, and silencing tools and techniques, such as CRISPR and RNA interference technology;

• Development, preclinical and clinical testing of non-viral and viral vectors for gene therapy;

• Cell Therapy strategies promoting neuroprotection and cell replacement in the CNS;

• Development, preclinical and clinical testing of adult and fetal stem cells, embryonic stem cells, and induced-pluripotent stem cells;

• Cell reprogramming strategies for the establishment of cell types to be used in the CNS;

• Advanced strategies for clinical translation in gene and cell therapy;

• New concepts for individual and precision medicine approaches in gene and cell therapy.