Tailoring Functional Nanomaterials for Efficient Gene Therapy and Tissue Regeneration
발표자
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초록
내용
Various gene delivery vectors, such as nanoparticles and recombinant viruses, have been developed for gene therapy in diverse tissue regeneration applications. Howerer, especially for the central nervous system (CNS), their practical application in clinical settings has been restricted due to the ineffective delivery of genes to target cells in the CNS. This inefficiency is primarily caused by the limited ability of vectors to penetrate the blood-brain barrier (BBB). Among these vectors, adeno-associated virus (AAV) is extensively utilized in numerous clinical studies owing to its favorable safety profile and relatively low immunogenicity compared with other viral vectors. Therefore, we aimed to develop novel AAV capsid variants with greater specificity to desired CNS tissues to enhance the gene transfer efficacy and reduce the unintended transduction in peripheral tissues including the liver and heart.