Roles of innovative genome editing technologies in stem cell engineering, rheumatic diseases and other joint/bone diseases

Gene editing also called genome editing, refers to a group of technologies that provide the possibility to modify the genome of a living organism. These technologies allow DNA to be added, removed, or altered at specific locations in the genome. Since the discovery and application with zinc-finger nucleases and transcription activator-like effector nuclease systems and then with clustered regularly interspaced short palindromic repeats (CRISPR)–Cas9, gene editing has become one of the most powerful research tools enabling the exploration and verification of disease-causing genes and variants. Genetic defects play a substantial role in the etiology of bone and joint diseases by interfering with either prenatal development of the skeletal system or postnatal bone and cartilage metabolism. Gene editing technologies have contributed to deciphering the mechanisms underpinning bone and joint diseases with their impressive application in cell and animal models. Recently, with the introduction of delivery strategies, gene editing, especially the CRISPR–Cas9, has shown great potential in disease modeling and gene therapy. In this chapter, we provide an overview of gene editing in prevention and therapy of bone and joint diseases through the engineering of specific elements of gene editing to the discovery of new delivery materials.