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British researchers to clone human stem cells to study motor neurone disease

BMJ 2005; 330 doi: https://doi.org/10.1136/bmj.330.7487.327 (Published 10 February 2005) Cite this as: BMJ 2005;330:327
  1. Kmietowicz Zosia
  1. London

The British research team that created Dolly the sheep has been granted a licence to clone human embryos to study motor neurone disease.

The licence will allow researchers at the Roslin Institute in Edinburgh and King's College in London to generate stem cell lines from embryos created using DNA from patients with motor neurone disease. They will focus on uncovering the cause of the motor neurone disease in patients whose condition cannot be linked to the genes already identified as causing the disease.

It is only the second time that the United Kingdom's Human Fertilisation and Embryology Authority has granted a licence for therapeutic cloning. The first licence was granted last year to the Newcastle Centre for Life, where researches will use the stem cells they generate to research diabetes, among other diseases (BMJ 2004;329:417).

Under the latest licence, the stem cells will be generated by first growing patients' skin or blood cells in the laboratory. The nucleus from one of these cells will then replace a nucleus from an unfertilised egg, which will then be allowed to grow to the “200 cell” stage. Growth factors will then be used to direct stem cells harvested from the egg to grow into motor neurone cells.

The technique, known as cell nuclear replacement, gives researchers a unique opportunity to investigate what causes the cells to degenerate in people with motor neurone disease.

“We will compare the behaviour and chemical profile of neurones with the gene defect to those without. The will tell us about the earliest events that ultimately lead to cell death,” said Professor Christopher Shaw from King's College, London.

The cultured neurones will also be used to test the ability of new drugs to halt or reverse the disease process. Hundreds of thousands of drugs can be screened in cultured cells in a year for about £100 000 ($186 000; €145), whereas it takes nearly two years and £20m to screen just one drug in patients.

“This is potentially a big step forward for MND [motor neurone disease] research,” said Professor Shaw. “We have spent 20 years looking for genes that cause MND, and to date we have come up with just one gene. We believe that the use of cell nuclear replacement will greatly advance our understanding of why motor neurones degenerate in this disease, without our having to first hunt down the gene defect.”

Professor Ian Wilmut from the Roslin Institute added: “This is not reproductive cloning in any way. The eggs we use will not be allowed to grow beyond 14 days. Once the stem cells are removed for cell culture the remaining cells will be destroyed. The embryonic stem cells that we derive in this way will only be used for research into motor neurone disease.”

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