What is Gene Editing?
Gene editing is a technology that involves making specific changes or modifications to the DNA sequence of living organisms for various research purposes. In gene editing, any DNA sequence of different organisms can be added, removed or corrected to treat acquired or genetic diseases.
Gene editing, also known as genome editing, works with the introduction of enzymes like nucleases that target a specific DNA sequence and help the scientists remove the existing DNA and add the required one by making a slit into the DNA strands. This can bring about changes in the organism’s traits such as eye color, hair color or disease susceptibility.
How does Gene editing technology work?
The most used gene editing tool is CRISPR/Cas9, short for clustered regularly interspaced short palindromic repeats. This Is a very effective genome editor that targets multiple DNA sequence sites, and it is an RNA-based system. The procedure of CRISPR/Cas9 is mimicked from the mechanism of bacteria when infected with a virus. Cuts made on the DNA trigger a repair mechanism by the cells to stitch the cut together.
This mechanism is utilized by CRISPR/Cas9. It slits a DNA sequence at a designated location to alter, add or delete the DNA. Cas9 is the enzyme responsible for cutting the DNA, and it is directed to the area of the DNA that needs to be altered by a strand of RNA. After this, the cell repair mechanism commences, and the cut is mended back together. Gene editing modification can be to a single base pair of DNA or larger sequences of chromosomes.
Read more: Genomic Technology and its applications
Applications of Genome editing technology
Gene editing, or genome editing, is still being explored and researched to ascertain its safety for use in humans. Gene editing is utilized overall for the treatment and prevention of diseases in animals or humans, whether acquired or genetic.
It is being researched for the treatment of complex diseases like mental illness, cancer, heart disease and HIV (Human Immunodeficiency Virus). Also, the treatment of diseases like sickle cell disease, diabetes, cystic fibrosis and hemophilia is being worked on using gene editing.
Animals have much the same genes as humans, so scientists experiment on animals, like mice, by editing their genomes and changing single or multiple genes to see how the changes affect the mouse. The results observed are then used to predict the outcome the changes will have when done on the human genome and how it is expected to affect human health.
Gene editing therapy is classified into two:
Somatic gene editing and Germline gene editing.
Germline gene therapy produces changes that can be transferred from generation to generation by making changes to the DNA of the reproductive cells, like eggs and sperm. While Somatic gene editing produces changes that manifest only in the individual receiving the treatment due to its target on non-reproductive cells.
Somatic gene therapy was successfully used in 2015 to treat a one-year-old in the United Kingdom suffering from leukemia. The gene editing tool used by the scientists for the treatment was TALENs.
Ethical Concerns and Implications of Gene editing technology
Some ethical concerns on gene editing are still being considered by the scientific community to properly ascertain its general use on the public.
- The safety of Gene editing and its technology to be used as a treatment procedure is still being explored.
- The cost of Gene therapy might be too high to be accessed by the entire public but only the rich.
- The use of Gene therapy on an embryo without the possibility of seeking permission before treatment.
- Germline gene therapy produces changes that are transferred to successive generations, therefore, the editing of germline cells is of concern for the unborn generations.
- The morals around some people who would want to use gene editing therapy and technology for acquiring traits that are not health-related such as height, athletic abilities, intelligence, hair, etc.
Germline genome editing brings up most of the ethical concerns in gene editing therapy, mainly due to the changes that pass on from generation to generation. To this effect, the editing of genes in eggs and sperm or of an embryo is currently illegal in many countries.