mouse-model
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Oral drug may change the story for huntingtin lowering
Researchers with PTC Therapeutics recently published exciting new findings – a promising new huntingtin lowering drug that can be taken as a pill. Will this change how we move forward with huntingtin lowering?
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GPR52: Exploring a new way to lower huntingtin
A Chinese research team developed a new way to lower huntingtin protein indirectly, by targeting a protein called GPR52. The molecules they designed were protective in cells and in mice with HD.
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Screening the entire genome for new drug targets for HD
Scientists screen the ENTIRE genome to find new potential therapeutic targets for HD. This ambitious study provides a wealth of data for HD researchers
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Could molecular handcuffs lower the protein that causes Huntington's disease?
Researchers got surprisingly lucky when looking for drug molecules to pull mutant huntingtin protein into a cellular garbage disposal machine
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Exciting new Huntingtin lowering tool described
Exciting new Huntingtin Lowering work from @SangamoTx and @CHDIfoundation using "Zinc Fingers" to shut down expression of the mutant Huntingtin gene. More details on this exciting new technique here.
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A step forward for gene editing: CRISPR-Cas9 and HD
Evolving CRISPR-Cas9 techniques can now be used to edit the HD gene in a living mouse brain.
By Dr Leora Fox -
Enemy at the gates – huntingtin disrupts nuclear transport
Two recent studies show how a cellular border control system goes wrong in HD, opening new avenues for HD research.
By Tom Peskett -
The benefits of migration, highlighted in Huntington's disease
Is HD a developmental disease? HD stops neurons migrating in developing brain, but maybe we can get them going again
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Early exposure to the HD protein may cause life-long symptoms
A surprising new mouse study suggests the mutant HD gene may do some of its damage during embryonic development
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Huntingtin takes a trip: harmful proteins pass between brain cells
Harmful misfolded huntingtin can travel between brain cells via messenger particles called exosomes
By Dr Leora Fox