Gene silencing could cure all diseases: scientists
Published: August 2, 2005
A new technique that shuts down gene expression at the DNA level could help cure all diseases, United States scientists say.
Mr David Corey, professor of pharmacology and biochemistry, and his team at the University of Texas Southwestern Medical Centre at Dallas shut down gene expression in cultured cells by blocking the ability of chromosomal DNA to be copied into RNA and made into proteins, reports science portal EurekAlert.
They targeted chromosomal DNA in two ways. First, they developed a synthetic molecule called a peptide nucleic acid, or PNA, which physically binds to DNA and blocks enzymes from copying or transcribing the DNA into messenger RNA.
More importantly, they also employed RNA itself as a silencing agent.
“Virtually every disease starts at the level of malfunctioning gene expression, or viral or bacterial gene expression,” Mr Corey said. “This is an approach that could theoretically produce a drug for the treatment or cure of almost any disease.”
Genes are segments of DNA housed in the chromosomes in the nucleus of every cell. Genes carry instructions for making proteins, which in turn carry out all of life’s functions. Faulty or mutated genes lead to malfunctioning proteins, which cause disease.
The information in a gene is not directly converted into proteins, but first is copied by special enzymes into many copies of messenger RNA, which then move out of the nucleus and into the body of the cell where they go on to create a protein.
Current techniques for turning genes on or off focus on controlling the messenger RNA once it’s already produced. But blocking all the copies of messenger RNA before they can make a protein within a cell is akin to using a bucket to catch all the streams of water coming out of a yard sprinkler before they can hit the ground.
Previous work by other scientists had shown that RNA might be able to target chromosomal DNA, so once Mr Corey and his team saw that PNAs were working, they decided to try RNA as well.
“The RNA is more important because it may reflect the body’s own natural mechanism for controlling gene expression, while the PNAs are synthetic,” Mr Corey said.
“The experiments worked beautifully,” he said. “It’s hard to believe that this strategy would work so well if nature wasn’t doing it already.”
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