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The private bank, which is the first of its kind in the world, includes 18 colonies of ES cells with rare genetic defects that trigger ten congenital diseases. Scientists will be able to grow these into human tissues that malfunction in the same way they do in patients with these disorders, providing unprecedented models for studying their development and testing promising new drugs.
The bank, opened by the Reproductive Genetics Institute in Chicago, also contains more than a hundred “lines” of normal human ES cells, almost doubling the number available to medical researchers. The cells are master cells found in embryos that can grow into any of the 220 types of tissue in the human body. They have great medical potential, because it should be possible to grow them into replacements for cells that malfunction in certain conditions, such as dopamine neurons in patients with Parkinson’s disease or insulin-producing islet cells in diabetes.
Ian Wilmut, who was awarded Britain’s second licence to clone human embryos last week, plans to use the ES cells he derives for this purpose and will study motor neuron disease.
He will produce cloned embryos from skin cells of patients with a genetic form of the wasting condition, strip out the ES cells and coax them to develop into defective motor neurons that can be examined for clues to the disorder and used to test drugs.
The institute’s bank has been established using embryos that were left over after a particular form of fertility treatment offered at its IVF clinic.
When a couple know they are carriers of genes that cause inherited diseases such as Huntington’s or beta-thalassaemia, they can opt to have children after screening IVF embryos for the condition using a technique called pre-implantation genetic diagnosis (PGD). A single cell is removed from each embryo and checked for the genetic defects involved, and only healthy ones are transplanted to the prospective mother’s womb.
The director of the Chicago institute, Yury Verlinsky, whose work is published in the journal Reproductive BioMedicine Online, has grown the ES cells in the bank from embryos screened out during PGD, which would otherwise have been destroyed.
Each of the ten diseases featured in the ES cell lines is routinely screened for by PGD. Three lines are available for Huntington’s disease, an incurable and fatal neurodegenerative disease, and five carry neurofibromatosis, which causes the growth of disfiguring tumours.
There are two lines with the defect that causes beta-thalassaemia, a form of anaemia, and two carry the muscle-wasting disorder myotonic dystrophy.
The other diseases, which each feature in a single cell line, are Duchenne muscular dystrophy, Becker muscular dystrophy, adrenoleukodystrophy (ALD), Marfan syndrome, Fanconi anaemia and Fragile X syndrome.
British scientists will have access to the cells through the institute’s offshoot in London, run by Mohammed Taranissi, of the Assisted Reproduction and Gynaecology Centre.
Patient groups welcomed the launch of the bank. Cath Stanley, of the Huntington’s Disease Association, said: “This is an exciting development in research into Huntington’s disease, and hopefully will bring us a step closer to a treatment or cure for this illness.” Sara Hunt of ALD Life, which supports families affected by adrenoleukodystrophy, said: “This is a good development in looking at the pathogenesis of adrenoleukodystrophy and making research more secure in the future.
“We very much hope it will help speed a cure for this cruel and devastating disorder.”
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