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17.6.11

Scientists change genetic code - Hope for treatment of genetic diseases

Scientists have discovered a way to change the genetic code, raising the possibility of future new treatments for genetic disorders such as cystic fibrosis, muscular dystrophy and several types of cancer.
The genetic code is the set of instructions in a gene that tell a cell how to make a specific protein.
When proteins are being synthesised by cells, a type of nucleic acid called messenger RNA (mRNA) carries the instructions from DNA and directs the steps necessary to build a protein.

However, mistakes can, and do, happen. Frequent mutations in DNA and mRNA can lead to flawed proteins that have the potential to cause serious harm.
The researchers at the University Rochester Medical Centre, New York, looked at a common type of mutation which can occur on mRNA molecules, known as a pre-mature "stop" codon. This causes a cell to stop reading the genetic instructions during protein production, which results in the formation of a shortened, faulty protein.

However, using another type of RNA molecule, the researchers were able to alter the mRNA in a way that turned the stop signal into a "go" signal, so that a normal full-length protein was formed.
Although the team produced the results both in a test tube and in live yeast cells, the hope is to use the technique in the body.

"This is a very exciting finding," said Yi-Tao Yu, an associate professor of Biochemistry and Biophysics, who led the study. "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."
Dr Robert Bambara, chair of the Department of Biochemistry and Biophysics, added: "The ability to manipulate the production of a protein from a particular gene is the new miracle of modern medicine.

"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."
The findings are published in the journal Nature.

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