ABSTRACT

In this chapter, the primary focus is on how genetic technologies are being applied to treat disease, but the authors begin by taking broader look at different treatment strategies for genetic disorders. They are primarily concerned with molecular approaches to treating disease, and deal principally with genetic approaches. Finally, they cover newer developments: RNA therapeutic approaches, therapeutic genome editing using programmable nucleases, and some new genetic approaches to preventing disease. The authors consider some novel genetic approaches aimed at preventing disease in the chapter using molecular genetic approaches to treat disease might involve many different strategies. Proponents of germ-line gene therapy argue that as genetic modification techniques become ever more refined, germ-line genetic modification could be used to eradicate a variety of severe genetic disorders, such as Huntington disease. Mitochondrial replacement has been used in mouse and primate models, with encouraging results, but at the time of writing, mitochondrial donation is still illegal in most countries, including the USA.