Folks,
Anybody have an opinion on gene therapy technologies and companies? Yesterday's article jogged my thinking on this ...
New U.S. approach to gene therapy found
WASHINGTON, March 3 (Reuters) - A new approach to gene therapy has worked in rats and may offer a better way to tackle genetic defects, researchers reported on Tuesday.
The new method, called chimeraplasty, was developed by Newtown, Pennsylvania-based Kimeragen Inc. A team at the University of Minnesota used it to change the genes of laboratory rats.
They targeted the liver cell gene for Factor IX, one of the blood proteins required for clotting. People with defects in the Factor IX gene suffer from hemophilia B, also known as Christmas disease.
"In this study we have targeted the Factor IX gene in animals and induced an alteration of that gene in the liver, the site of clotting factor formation, and produced animals with a mutated clotting factor as predicted," Dr. Clifford Steer, a professor of medicine and cell biology at Minnesota, said in a statement.
"Our research team, through a series of experiments, has demonstrated that this new class of molecules, chimeraplasts, can be designed to target specific sites within the genes and efficiently alter an animal's DNA."
The method takes the desirable stretch of DNA and combines it with RNA, which is the chemical that translates DNA's genetic code into something the body can actually use -- a protein.
Scientists sometimes describe DNA as the recipe, RNA as the cook and the protein as the finished dish.
The DNA plus RNA molecule is called a chimeraplast. The method avoids the use of a vector -- usually a virus -- to carry the gene into the cell.
Using viruses can cause an immune response and is an imprecise method of delivering genetic material.
Chimeraplasty lets scientists target precisely on which gene and where the new stretch of DNA will go.
"This is the breakthrough in gene therapy that I have been waiting for," said Dr. Michael Blaese, chief scientific officer for Kimeragen.
"It is our intention to correct hemophilia A and B in animals and then work with the FDA (the U.S. Food and Drug Administration) on human trials beginning as early as 1999," said Dr. Gerald Messerschmidt, president and chief executive officer of Kimeragen.
21:42 03-03-98
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