Image courtesy of DepositPhotos
Scientists use gene-altering technologies for many purposes. These technologies can change a person’s DNA to alter physical traits. For example, they might use technology to change a person’s eye color or lower their disease risk. The technologies target the DNA at a specific site. With their use, scientists can add, remove, or replace DNA.
Sickle Cell Anemia
Individuals with sickle cell anemia are grateful for gene editing innovations because they say CRISPR is life-changing. Humans have genes that make hemoglobin. When they have two bad copies of one of these genes, they develop this condition, which causes intense pain. A person with sickle-cell anemia has an average life span of 53 years, but that may change. Medical professionals edit the DNA in bone marrow cells to turn a dormant gene capable of making hemoglobin back on. Most people don’t know much about gene editing, but with treatments like this on the horizon, they soon will, thanks to alternatives to CRISPR being developed.
The History of Gene Altering
Gene-editing technologies have existed since the late 1900s. However, it wasn’t until 2009, when CRISPR was introduced, that editing DNA became simpler and faster. This innovation is also cheaper and more accurate.
How Gene Editing is Used
Scientists use gene editing for many purposes. For example, they alter genes to learn more about different diseases. When doing so, they typically use mice or zebrafish, as both species share many characteristics with humans. Scientists can alter one or more genes in a mouse to see how humans might react to a drug. Humans and mice share 85 percent of the same genes, so researchers expect humans to respond to a drug or treatment like humans. National Human Genome Research Institute (NHGRI) scientists are researching zebrafish genomes to learn more about human deafness, and this is one of many examples of how gene editing technologies might benefit humans.
New Treatments
Researchers are developing different treatments using gene editing therapies. They believe these therapies may help individuals diagnosed with diabetes or cystic fibrosis much as they are helping those with sickle cell anemia. Germline therapies focus on changing the DNA in reproductive cells so these alterations can be passed down to future generations. Somatic gene-altering therapies work on non-reproductive cells and only impact the individual.
Somatic gene editing technology was used to treat a one-year-old girl in 2015. This young lady was diagnosed with leukemia and received a gene editing treatment known as TALENs. Doing so saved her life, but this treatment remains experimental, as medical professionals and policymakers want additional information before offering this treatment option to the general public.
Gene Editing Challenges
When using gene editing tools, care must be taken, as they may cut in the wrong spot. Scientists continue to research why this is happening and how to avoid it. In addition, other steps must be taken to ensure the safety of these therapies before using them on humans.
Ethical Considerations
Scientists must recognize that gene editing comes with ethical concerns. Safety must be the top priority, and scientists are debating whether it should be used on embryos because they can’t give permission. Currently, the NIH doesn’t fund research involving gene editing of embryos.
They also need to figure out how to make this technology affordable for all and when it should be used for procedures that will improve a person’s physical characteristics for reasons other than health. Finally, scientists must decide when it would be appropriate to edit germline cells, as these alterations would impact future generations.
Gene editing innovations will continue. Using these innovations ethically is a concern, but research must continue, as these innovations may be used to cure diseases that affect countless people. When done correctly, this research could change the world.
