By Dr Jeff Carroll May 07, 2019 Edited by Dr Tamara Maiuri
Today saw the publication of the first manuscript describing a huntingtin lowering trial in Huntington’s disease patients. This study, sponsored by Ionis and Roche, provided clear evidence of researchers’ ability to safely reduce mutant huntingtin protein in the spinal fluid. An overview of these results has previously been shared, but this manuscript provides important new information about the results of this remarkable trial. What’d we learn?
Huntingtin lowering: a little background
Huntington’s disease (HD) is caused by a single mutation in a gene we refer to as the HD gene. Like most genes, the HD gene is used by cells as instructions to create a little cellular machine called a protein, which we call huntingtin. The genetic mutation that causes HD also changes this protein, which we refer to as mutant huntingtin.
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