stem-cells
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First participant dosed in pioneering neural stem cell trial for Huntington’s disease
7 min read
A new clinical trial for HD is underway. The first participant has been dosed in REGEN4HD, a study testing whether transplanting neural stem cells is safe as a potential treatment for HD.
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Turning down mismatch repair genes slows Huntington’s repeat growth in human neurons
9 min read
In a human cellular system, scientists dialed back mismatch repair genes to reduce CAG expansion by up to 69%. This work is in its early days, but it shows promise for “anti-expansion” therapies that could be used to delay HD onset.
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Tooth fairy therapy? Stem cells from teeth tested in a small Huntington’s disease trial
8 min read
A trial tested stem cells from human teeth as a possible treatment for HD. The approach appeared safe with some improvements in symptoms, but the small size of the study and the unclear underlying science of this approach means caution is urged.
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Energy off balance: How Huntington’s disease influences the cell’s powerhouse
8 min read
Scientists used 3D mini-brains grown from stem cells to study Huntington’s disease. They found early developmental changes linked to mitochondrial stress, suggesting that energy imbalance may play a role in HD, even before neurons fully form.
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Replacing What Is Lost: Regrowing Damaged Brain Cells for Huntington’s Disease
7 min read
Cell replacement therapy is making massive strides for other diseases. But what about Huntington’s disease? Can we use technological advancements in this area to replace lost brain cells with the hope of repairing and reversing disease?
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Mini brains grown in a dish shed light on Huntington’s disease and how we might treat it
7 min read
Exciting new findings using 3D human lab-grown mini brains inform ongoing HTT-lowering trials and suggest that stem cell transplants for HD may improve cell-to-cell communication and reduce disease features.
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The VIP section: velvet ropes for the brain and how to get in
6 min read
New work from two groups advances tools to model the blood-brain barrier and could improve the way drugs are delivered for brain diseases. This builds on our ability to study, and eventually treat, Huntington’s disease.
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Youthful competitors: young brain cells oust the old
5 min read
Replacing cells with HD in the brain could be an effective treatment strategy. Recent work shows that glia injected into mouse brains take over and oust the older cells, but for a surprising reason – because of age, not HD!
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The third dimension: using minibrains to understand brain development changes in HD
10 min read
Researchers show that highly expanded CAGs in the HD gene can cause early developmental changes using 3D brain models called organoids. What’d they find?
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Huntington's disease therapeutics conference 2018 – day 1
7 min read
Our daily roundup of the science presented at the 2018 HD Therapeutics Conference in Palm Springs