Brain-targeted delivery of CRISPR/Cas9 mediated glioblastoma therapeutics

Abstract

Glioblastoma multiforme (GBM) is a life-threatening malignant tumor of the central nervous system, for which there is currently no effective treatment. Its low survival rate has been interpreted as a result of its high proliferation rate, resistance to apoptosis, and the ability to create a microenvironment conducive to tumor growth. Recently, a powerful and accurate gene-editing tool, Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-Associated Protein 9 (CRISPR/Cas9), has high potential in various scientific fields. Such technologies can manipulate cellular mechanisms and defective genes that lead to the progression of many serious diseases such as cancer. One of the major barriers to the application of this technique is the development of a delivery method to diffuse CRISPR/Cas9 efficiently and accurately to the target location in brain. Most existing delivery methods are failed to be translated into clinical result due to the lack of promising safety and efficiency. Thus, I will introduce several strategies here that can be used potentially for CRISPR-Cas9 system delivery in GBM treatment including the principle, advantages, limitations, and latest developments of these systems. This review is composed to provide a concise summary for future researchers to understand the current challenges and approaches in CRISPR/Cas9 mediated GBM therapeutics delivery.