Genome Editing and Human Health
The potential of genome editing to improve human health is only beginning to blossom. For example, CRISPR-Cas9 has been used as an approach to attack antibiotic-resistant bacteria (Waddington et al., 2016). Research involving genome editing has been used to address currently untreatable genetic diseases such as Duchenne’s muscular dystrophy, as well as human pathogens, such as HIV and hepatitis B virus (Yin et al., 2014, Benjamin et al., 2016, Mendall et al., 2016).
Today, genome-editing studies have been conducted using cell culture and animal trials, including non-human primates, to realize authentic changes to disease status (Niu et al., 2014, Stone et al., 2016, Wang and Qi, 2016, Zhou et al., 2016). For example, the genetic disease cystic fibrosis (CF) can potentially be eliminated by genome editing and has been shown to work so far both in human cell culture as well as in a mouse model. The defective gene involved in CF can be corrected in inducible pluripotent stem cells, indicating that this genetic disease could be cured before its onset and removed forever from subsequent generations. Direct correction of the mutation in adult diseased lungs is also under consideration. While corrections may not reach every single epithelial cell in the lung of an infected patient, the resulting mosaic of edited versus unedited cells may still be sufficient to greatly reduce or eliminate symptoms of the disease (Alton et al., 2016).
Genome editing could also be used in the future to treat hereditary movement disorders, including Huntington’s and Parkinson’s disease (Seah et al., 2015, Im et al., 2016). For example, deletion of the defective gene that is responsible for Huntington’s disease in mice has been shown to prevent protein aggregation in the brain and thus disease symptoms (Talan, 2015). Furthermore, genome editing may play a significant role in a variety of forms of cancer therapy (Yi et al., 2016). The fate of patients with difficult to treat mitochondrial diseases could potentially be improved using genome editing technologies (Fogleman et al., 2016). Some researchers believe that genome editing could offer improvements in medicine that have never been realized before. As of now, the technology is too new for adequate appraisal either of potential or of social implications (Singh et al., 2016). Who will decide? Who will govern?