Researchers are working on new ways to cure diseases such as Alzheimer’s disease, blindness and cystic fibrosis using CRISPR-Cas9 technology

CRISPR technology is constantly evolving; it deals with finding and altering DNA inside a cell. Experts believe CRISPR is a promising tool for the future of cancer therapies and curing life-threatening diseases such as Hypertrophic cardiomyopathy, a fatal heart condition.

CRISPR-Cas9 technology is an accurate way of editing cells, tissues and whole organisms. Currently, CRISPR systems are being used to treat genetic disorders in animals and soon will be used to treat human diseases of the eye and blood. In China and the United States, the use of CRISPR-Cas9 to treat targeted cancer has been approved after clinical trials have taken place.

CRISPR-Cas9 technology is being used by scientists to edit the human genome. Recently, there has been proof that CRISPR-based genome editing can correct faulty genotypes. This means mutations that cause Huntington’s disease or cystic fibrosis, or the BRCA-1 and 2 mutations linked to breast and ovarian cancers, can potentially be edited using CRISPR-Cas9. For example, a cystic fibrosis patient had the transmembrane regulator sequence edited which resulted in stem cells differentiating into airway epithelial cells. CRISPR-Cas9 technology can also be used on animals such as in rats, where a rhodopsin mutation was corrected by injecting plasmids encoded by CRISPR-Cas9.

While this is a major advancement in CRISPR-Cas9 technology, scientists are urging caution on human testing. This is because the Cas9 enzymes could potentially cause harm or create new diseases in human cells if they edit DNA in the wrong places. Due to this reason, Jiankui’s experiment in producing human babies with CRISPR-edited genomes had caused an outrage amongst fellow scientists.

In 2018, Jiankui He, an associate professor from Southern University of Science and Technology, announced two babies with edited CCR5 genes had been born in China, and these babies would be immune to HIV infection. The evidence of this experiment that had been revealed by He showed that it was wrong on scientific and ethical levels. Researchers working in the same field were shocked by this as they believed editing in embryos is not necessary to prevent HIV transmission to the foetus. Hence causing scientists worldwide to develop criteria and standards for genome editing in humans, ensuring scientific and ethical morals are kept in regard when performing these experiments.

As the world population is increasing, CRISPR technology is also starting to play a major role in food enhancement. It has important implications for improved worldwide agriculture and food security. CRISPR-Cas9 technology could increase the quality and shelf life of the crops, making the crops insecticide resistant and improve food safety.

The concept of bioengineering food is not new, it is simply evolving with CRISPR-based systems. Scientists are hoping for a future where CRISPR-Cas9 technology develops climate-resilient and disease-resistant plants; a future where breeding livestock could be replaced permanently by CRISPR-based genome engineering; a future where CRISPR-Cas9 technology could be the cure to diseases like Alzheimer’s disease. Now, CRISPR-Cas9 technology has provided researchers with a quick and easy way for genome editing – in days rather than weeks or months. Soon CRISPR technologies will be able to study immune systems and how diseases are transmitted in large animals.

References:

Barrangou, R. and Doudna, JA. (2016). Applications of CRISPR technologies in research and beyond. Nature biotechnology, 34(9), pp. 933-941.

Haskell, S. (2020). CRISPR and Our Food Supply: What’s Next in Feeding the World? Michigan State University.

Plumer, B., Barclay, E., Belluz, J. and Irfan, U. (2018). A simple guide to CRISPR, one of the biggest science stories of the decade. Vex.

Tangermann, V. (2018). A CRISPR Future: Five Ways Gene Editing Will Transform Our World. Futurism, 30 January.

Wang, H. and Yang, H. (2019). Gene-edited babies: What went wrong and what could go wrong. PLOS Biology, 17(4).