To What Extent Will Genome Editing Become an Available Cure for Genetic Disease?

Abstract

Genome editing has many potential applications for the treatment of genetic disorders. Following a review of the literature, an overview of genome editing technologies, ZFNs, TAL-ENs and CRISPR-Cas9, is presented. The use of CRISPR-Cas9 systems to develop treatments for hemoglobinopathies, including sickle cell, is outlined as well as the potential for the technology to target more diseases such as muscle dystrophy. This type of treatment and the ethical considerations that come with introducing genome-altering technologies for clinical use are explored in this article. A judgment is then drawn that it is likely genome editing will be an available cure for genetic diseases, however, restrictions and regulations against the misuse of this valuable remedy must be put in place to ensure the conservation of the human genome.

Keywords: Genome editing; CRISPR; Ethics; Sickle cell disease; Duchenne muscular dystrophy