Gene editing is the process of making tiny and controlled changes in the genetic material of a living organism. Gene editing technologies raises hope for understanding the fundamental cause of genetic diseases and possibly how to repair them.
Recently, researchers published a study in the journal Nature about their use of new gene-editing technology in mice that provide a model for the premature aging disorder known as Hutchinson-Gilford Progeria Syndrome.
Most of the premature aging disorders are due to problems in DNA maintenance. Unfortunately, there is no cure yet for the premature-aging disorder progeria syndrome. But could this new gene-editing tool offer a way to treat the condition?
Base Editing Repairs Mutation of Progeria Syndrome in Mice
The researchers have shown that they can correct the single-letter genetic mistake that causes thousands of genetic diseases and create healthier and longer living mice.
The study's senior author, David Liu, said that there is still a long road ahead for the gene-editing tool called base editor can be used to treat genetic diseases but establishing it can correct a mutation that causes devastating diseases in an animal to rescue them from the symptoms of the disease.
Liu and his colleagues repaired a gene in mice, that if it mutates it could cause progeria syndrome, USA Today reported. On average, people suffering from progeria syndrome only live by just 14 years and die of health conditions, like heart diseases, which is the common disorder that kills older people.
Unlike previous technologies of gene-editing, base editing works like a pencil with an eraser that erases the single-letter genetic mistake and rewrites it. In the past, gene-editing technologies work like a scissor that cuts the double-strand DNA. But Liu said that the method for treating genetic diseases should be precise rather than disrupting.
Gene editing expert Fyodor Urnov from the University of California-Berkeley, who is not part of the study, said that the base editing technology is a huge scientific achievement. But he noted that there is a long way to go before the same technology can be used in fixing the genetic diseases in a person.
According to the report in Nature, if base editing is used on humans, their safety must be ensured.
Genetic Engineering Is Improving
The field of genetic engineering is improving and moving forward in recent years with the discovery and creation of the CRISPR-Cas9 a few years ago and also the base editing technology that can repair mutations of progeria syndrome in mice.
However, like many great inventions, it is not without failures and controversies. The biggest controversy in genetic engineering happened in 2018 when Chinese scientist Jiankui, who is behind bars now, gene-edited embryos, which led to the birth of three babies. He used the CRISPR-Cas9 method in his experiment.
CRISPR method worked effectively in humans in the past year. CRISPR was used in the eye to treat a genetic form of blindness. Additionally, a sickle cell patient and another one with blood disorder beta-thalassemia were treated by CRISPR last month. The patient with sickle cell has avoided pain crises, while the latter stopped regular blood transfusions last year.
CRISPR-Cas9 is used as a starting point for Liu's base editing gene-editing technology.
READ MORE: Scientists Warn Human Genome Editing Might Introduce Unwanted Genetic Defects
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