Advances in Molecular Biology in Progression Towards Human Genome Engineering Therapies

Abstract

While genetic disorders are individually rare, many exist, and collectively these conditions affect millions of people. The lack of available treatments and cures for these conditions has resulted in hundreds of millions of dollars spent in caring for these patients each year globally. Gene therapy techniques were a first-pass attempt at curing these diseases; clinical trials for gene therapy increased dramatically throughout the 1990s into the 2000s. Gene therapy methods, however, were risky due to 1) adverse effects genetic vectors used had on patients and 2) a lack of knowledge of the human genome and the adverse effects foreign DNA expressed in humans might have. Current advances to both viral and non-viral genetic vectors have reduced immunogenicity and increased delivered DNA stability in host tissues. Next generation sequencing has enabled rapid, inexpensive, and accurate whole-genome sequencing that is very useful for not only learning more about the human genome and genetic disorders but also for providing patients better and more targeted care in the clinic. The development of CRISPR/Cas9 genome editing methodologies have revolutionized the possibly surrounding genome therapies in humans. These dramatic improvements in molecular biology, genomics, and bioengineering research all point to a revolution in modern medicine.