'Miracle' Treatment for Genetic Diseases Discovered by Scientists

Published June 16, 2011

| NewsCore

A new technique to alter the genetic code behind hundreds of diseases was hailed by U.S. scientists as a "miracle of modern medicine."

Genes responsible for devastating diseases can be corrected by controlling faulty messenger cells that are central to the body's protein production, researchers said Wednesday.

"The ability to manipulate the production of a protein from a particular gene is the new miracle of modern medicine," according to Dr. Robert Bambara, from the University of Rochester Medical Center. "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 researchers focused on genetic material called messenger RNA (mRNA) that can affect the production of vital proteins when a premature "stop" or "codon" signal occurs. This causes a cell to stop reading the genetic instructions partway through the process, resulting in the creation of an incomplete, shortened protein.

Writing in the journal Nature, the research team said that it produced normal, healthy proteins in test tubes and in live yeast cells by using short strands of another type of mRNA to correct the faulty ones.

The researchers were hoping to apply the same principles to the human body, with the aim of altering the course of genetic disorders such as cystic fibrosis, muscular dystrophy and some cancers.

"This is a very exciting finding," lead study author Professor Yi-Tao Yu 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."

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