Since the discovery of CRISPR endonucleases, the ability to permanently modify the DNA of a living organism has been a long-term goal of biotechnology.
In humans, gene editing has enormous promise as a potential cure to genetic diseases and serve as personalized medicine for a wide variety of medical conditions. A classic example of this is Duchenne muscular dystrophy (DMD). Children born with DMD have a mutation in the dystrophin gene that leads to fatal decay of skeletal muscle tissue. If the mutated dystrophin gene could be fixed with gene editing, the condition could be permanently cured.
Gene editing tools can be also used in genetic engineering to facilitate development of organisms with desirable characteristics. One recent example is an engineered bacterium that can break down plastics in the environment, enabling better breakdown of garbage in landfills and removal of plastic in the ocean.
However, the development of gene editing tools for medicine is still an emerging field. Many current experimental gene editing tools require breaks in DNA to insert modifications and there are a number of possible off-target effects that deserve to be studied. There are also substantial ethical questions and concerns about gene editing in humans; for example, what if parents decided to “edit” their child’s genes for more desirable characteristics?
Our research on gene editing is focused on the ability to write whole genes into the genomes of mammalian cells using programmable retrotransposons. A major priority of ours is studying the off-target effects and consequences of genetic insertions in the target DNA, so that we may better understand the context in which gene insertions occur.