Scientists Discover New Way to Treat Genetic Disorders

In a new study, scientists have discovered an entirely new way to change the genetic code, by which the course of devastating genetic disorders can be altered.

The findings may help researchers find treatment for genetic disorders, such as cystic fibrosis, muscular dystrophy and many forms of cancer.

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The genetic code is the set of instructions in a gene that tell a cell how to make a specific protein.

Central to the body's protein production process is messenger RNA, or mRNA, which takes these instructions from DNA and directs the steps necessary to build a protein.

For the first time, researchers artificially modified messenger RNA, and in doing so changed the original instructions for creating the protein. The end result: A different protein than originally called for.

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"The ability to manipulate the production of a protein from a particular gene is the new miracle of modern medicine," Robert Bambara, Ph.D., chair of the Department of Biochemistry and Biophysics at the University of Rochester Medical Center, said.

"This is a really powerful concept that can be used to try to suppress the tendency of individuals to get certain debilitating, and sometimes fatal genetic diseases that will forever change their lives," he said.

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In the study, researchers focused on a common type of mutation that occurs when an mRNA molecule contains a pre-mature "stop" signal, known as a pre-mature stop codon.

A premature stop codon orders a cell to stop reading the genetic instructions partway through the process, resulting in the creation of an incomplete, shortened protein.

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Researchers were able to alter mRNA in a way that turned a stop signal into a "go" signal. As a result, the cell could read the genetic instructions all the way through and create a normal, full-length protein. The team produced these results both in vitro and in live yeast cells.

"This is a very exciting finding," Yi-Tao Yu, Ph.D., lead study author and associate professor of Biochemistry and Biophysics at the Medical Center, said.

"No one ever imagined that you could alter a stop codon the way we have and allow translation to continue uninterrupted like it was never there in the first place," Yu added.

The study has been published in the journal Nature.

Source-ANI