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Inheriting small mutations can sometimes have devastating effects on people's lives. Otago Department of Biochemistry researcher Dr Stephanie Hughes and her research team study a rare genetic neurodegenerative disorder called Batten disease. They are developing a treatment using gene therapy, with the help of some sheep that also have the disease.

Click through the pages below to find out more about Batten disease and Dr Hughes's research to develop a treatment.

Download a pdf file of this story on Batten disease gene therapy here (3.7MB).

Picture of a young man with Batten disease in a wheelchair.

What is Batten disease?

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What causes Batten disease?

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Recessive genetic disorders, family trees, and genetic testing

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Gene therapy - a cure for Batten disease?

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Testing gene therapy for Batten disease in sheep

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Implications of gene therapy

More information on Batten disease and gene therapy:

NZ High School Biology

This research story is useful to New Zealand high school students studying NCEA Biology, particularly within these standards:

  • 2.7 Demonstrate understanding of gene expression
  • 2.4 Demonstrate understanding of life processes at the cellular level
  • 3.2 Integrate biological knowledge to develop an informed response to a socio-scientific issue
  • 3.7 Demonstrate understanding of human manipulations of genetic transfer
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