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Scientific Discovery Makes Drug More Accessible

by Medindia Content Team on Feb 5 2008 7:48 PM

Using gene therapy, researchers at University of Pennsylvania have decreased the time taken to breed large animals that produce therapeutic proteins in their milk such as insulin or proteins needed to fight cancer.

This can be a major scientific breakthrough in pharmaceutics, as existing methods involve cloning, involving more time and more money.

“Having an easier way to harness nature’s power to produce large quantities of specific proteins in milk could increase the availability of drugs for people who could otherwise not afford these treatments,” said Ina Dobrinski, one of the researchers on the study.

The study holds a lot of importance as it may also be a new way to eradicate diseases in future generations of animals, like those used for livestock.

The scientists, in order to get the goats to produce specific proteins, used radiation to kill a portion of a male goat’s germ cells (the cells that produce sperm).

After that they used a modified adeno-associated virus (a well studied and tolerated gene therapy vector) to put in a gene in the remaining cells. After the new gene took hold in the germ cells, an expected number of female offspring produced the desired protein in their milk.

Though, this scientific advance is instantly valuable for pharmaceutical development and biology research, a similar approach may also boost the food supply by eliminating genetic disorders in animals over several generations.

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Once perfected, this technique could also eradicate disease genes in humans over several generations, assuming ethical concerns can be resolved adequately.

“For thousands of years, people have domesticated cows and goats to make milk, butter and cheese. And for thousands of years dairy products have been used as folk remedies for practically every human illness. Most have been completely ineffective.” said Gerald Weissmann, MD, editor-in-chief of The FASEB Journal.

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He added: “So it is reassuring that modern science would find a way to use the milk we drink to yield of drugs that actually work.”

The research is published in the latest print edition of The FASEB Journal.

Source-Eurekalert
KAV/M


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