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. Last Updated: 07/27/2016

New Science Could Bring Novel Cures

ROSLIN, Scotland -- Cloning adult animals could eventually offer a range of benefits, from a cure for mad cow disease to effective new treatments for hemophilia, the researchers who pioneered the technique said Tuesday.


Herds of animals could now more easily be bred to produce milk, blood and organs used in a wide variety of medical treatments -- but such possibilities are still years away, they said.


"There are some scientists for whom it is just pure knowledge," said Ian Wilmut, who together with colleagues at the Roslin Institute near Edinburgh have made scientific history by successfully cloning an adult sheep. But there are also commercial benefits -- and these are the aim of Wilmut's group, which works with biotechnology company PPL Therapeutics.


PPL already breeds genetically engineered animals that are used to produce human proteins. It is clinically testing a product known as AAT (alpha-1 antitrypsin), a protein derived from the transgenic sheep's milk that they hope can be used to treat cystic fibrosis.


They are also working to create animals whose blood or milk produces human chemicals such as fibrinogen and Factor IX, important for blood clotting and of potential use in surgery and hemophilia treatment.


"The immediate application is in biotechnology, particularly in the production of pharmaceutical proteins," Wilmut said. "In the longer term there is the possibility of doing this in pigs for organ transplant."


The pigs must be genetically engineered so the human immune system will be tricked into accepting their organs. The creation of a perfectly transgenic pig would allow doctors to clone it and farm organs from its genetic twins.


The technology will also offer better opportunities to study scrapie and related diseases such as bovine spongiform encephalopathy (BSE, or mad cow disease), Wilmut said. BSE, scrapie and the human Creutzfeldt-Jakob Disease all have genetic components.