Understanding Genome Editing
Our DNA is at the root of each of us – that unique combination of genes that makes us all individuals.
Different sequences within our DNA dictate different characteristics; however, sometimes there are mistakes in DNA – mutations in genes that can cause many different kinds of serious diseases. Our DNA is at the root of each of us – that unique combination of genes that makes us all individuals. Familiar examples of genetically defined diseases include cystic fibrosis, Duchenne muscular dystrophy, Huntington’s disease, and sickle cell anemia.
Major investments in the human genome project and the subsequent development of low-cost and rapid DNA sequencing and informatics tools have revolutionized the understanding of genetically defined diseases and paved the way for advancing the field of genomic medicine.
Genomic medicine harnesses the knowledge of genetics to guide the care of patients and create new treatments.
There are several technologies that have the potential to create medicines in this field. These technologies can be grouped into two broad categories: gene augmentation and genome editing. Each approach seeks to address genetically defined diseases at the level of DNA. However, gene augmentation, which is commonly called gene therapy, and genome editing differ fundamentally with regard to the kind of genomic change they seek to accomplish.
Gene therapy is an approach whereby a new gene is transferred into cells to augment a defective gene. However, gene therapy does not remove or modify the defective DNA and is generally suited for a limited set of genetically defined diseases. Genome editing, in contrast, corrects the defective DNA in its native location. The diversity of genetic drivers of disease demands a diversity of solutions. Genome editing has the potential to deliver a variety of types of genome modification to address a broad range of genetically defined diseases.
Of the estimated 6,000 diseases that are caused by genetic mutations, more than 95 percent have no approved therapeutic option.
Where options do exist, they often only treat the symptoms of the disease or modify the course of disease, but do not address the underlying genetic defect.
6,000 diseases and only 5% approved therapies