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  Dolly creator to study ALS
Posted September 29, 2004 in ALS Research

Copyright 2004 Associated Press
All Rights Reserved
Associated Press Online
September 28, 2004 Tuesday
SECTION: INTERNATIONAL NEWS
LENGTH: 687 words
HEADLINE: Dolly Creator Applies for Cloning License
BYLINE: EMMA ROSS; AP Medical Writer
DATELINE: LONDON

The creator of Dolly the sheep, the world's first mammal cloned from an adult, applied for a human cloning license Tuesday to study how nerve cells go awry to cause motor neuron disease.

Ian Wilmut, who led the team that created Dolly at Scotland's Roslin Institute in 1996, said he plans to clone cells from patients with the incurable muscle wasting disease, derive stem cells from the cloned embryo, make them develop into nerve cells and compare their development with nerve cells derived from healthy embryos.

Such work, called therapeutic cloning because it does not result in a baby, is opposed by abortion foes and other biological conservatives because researchers must destroy human embryos to harvest the cells.

The United Nations is scheduled to vote on a convention on human cloning next month. The United States is pressing for it to ban all forms of human cloning, while Belgium heads a faction seeking a ban on reproductive cloning but the option for countries to approve therapeutic cloning.

Britain became the first country to legalize research cloning in 2001, but only now are scientists ready to start using the technique. They hope it will revolutionize medicine by providing better treatments for a variety of illnesses.

Wilmut said he hopes for a response from the fertility regulators early next year and to begin work in the spring.

The Human Fertilization and Embryology Authority, Britain's fertility regulator, granted the country's first license for human cloning last month to a team at Newcastle University that hopes to create insulin-producing cells that could be transplanted into diabetics.

But the latest project does not involve transplanting genetically matched stem cells, particularly challenging in motor neuron disease because the cells are so big and have to develop and extend their cable-like projections long distances to connect with muscles.

The mechanism behind motor neuron disease is poorly understood because the nerves are inaccessible in the brain and central nervous system and cannot be removed from patients.

"At the moment we have to rely on post-mortem tissue and that's very much the end stage," said Dr. Brian Dickie, director of research development at the London-based Motor Neuron Disease Association. "What we don't have, and what we desperately need, is a way to study human motor neurons as the disease progresses.

"If we're going to develop a treatment, the chances are that we are going to have to catch the disease at a relatively early stage," said Dickie, who is not connected with the research.

Motor neuron disease is a collection of illnesses that all lead to loss of muscle function because of nerve failure. About 350,000 people worldwide are afflicted and about 100,000 die every year.

About 10 percent of those stricken live for a decade or more, like celebrated physicist Stephen Hawking, who has a type of motor neuron disease called amyotrophic lateral sclerosis, or ALS, also known as Lou Gehrig's disease.

However, most die within five years of the onset of symptoms, which usually start in middle age.

An inherited defect in a single gene is responsible for about 2 percent of cases. Another 8 percent are caused by an as yet unidentified, inherited genetic abnormality.

Stem cells are the master cells of the body that go on to become every bone and tissue.

Wilmut and his colleagues plan to produce three groups of stem cells.

The first group would be regular stem cells extracted from a healthy donated embryo. The scientists would then take some of those cells and inject them with the damaged gene to make them develop the disease.

To obtain the third type of cells, they would clone a blood or skin cell from a patient whose motor neuron disease is inherited but caused by unknown genetic abnormalities. The resulting cloned embryo would then be mined for its stem cells.

All three groups of cells would be induced to grow into neurons and scientists would track and compare the development of the healthy neurons, the neurons with a single gene defect and those with the unidentified genetic problem.

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