mouse-model
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Could rewiring the brain’s “brakes” improve movement in Huntington’s disease?
6 min read
Scientists discovered that a specific type of brain cell becomes unusually quiet in Huntington’s disease. By restoring the activity of these cells in mice, they were able to rebalance brain circuits and improve movement.
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Taking A Short Cut: New Routes to Huntingtin-Lowering Therapies
6 min read
New research points to HTT1a, a toxic shortened form of huntingtin, as a key driver of disease and a promising therapeutic target in HD.
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Precision Pruning with PROTACs: Selective Targeting of Toxic Huntingtin
6 min read
PROTACs act like a precision pruning tool, trimming away toxic huntingtin protein while leaving the healthy form intact. In cells and HD mice, this targeted clean-up improved health and slowed disease features.
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Sealing the gap: a case for slower, smarter DNA repair
6 min read
Your body assembles DNA like a team of workers who set and bond a pattern of tiles. A recent study shows that one special assistant might slow somatic CAG expansion and delaying HD onset.
By Dr Chris Kay -
Fixing the Recipe: Lowering a Slice of Huntingtin
8 min read
In a recent paper, scientists targeted a small, harmful piece of the huntingtin message (HTT1a) in HD mice. This reduced toxic protein clumps and delayed genetic changes, more than targeting full length HTT.
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Tagging the Trash: Turnover of Toxic Huntingtin
5 min read
A recent study suggests that strengthening the brain’s own clean-up system could slow Huntington’s disease.
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Cutting to the chase with CRISPR
6 min read
A new study uses “self-switching” genetic scissors to target the root cause of Huntington’s disease in mice – even after symptoms begin.
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Unsung Heroes: Could Glial Cells Treat Huntington’s Disease?
7 min read
Transplanting healthy human glial cells into HD mouse brains improved movement, memory, and survival. Even more strikingly, the glia coaxed diseased neurons to behave more like healthy ones, offering a potential new path for treating HD.
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Knockouts for the win: how expanding CAGs drive disease
9 min read
A recent paper links CAG repeat expansion with widespread RNA changes, especially in brain cells vulnerable to HD. “Knocking out” certain DNA repair genes that go awry in HD can have positive effects on features of HD in mice.
By Dr Leora Fox -
How many is too many? Exploring the toxic CAG threshold in the Huntington’s disease brain
6 min read
New work from researchers in London uses mice to narrow in on the number of CAG repeats needed to cause symptoms of Huntington’s disease. Their work points to fewer than 185 CAGs as a threshold.
By Dr Chris Kay