mouse-model
-
Knockouts for the win: how expanding CAGs drive disease
A recent paper links CAG repeat expansion with widespread RNA changes, especially in brain cells vulnerable to HD. āKnocking outā 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 Huntingtonās disease brain
New work from researchers in London uses mice to narrow in on the number of CAG repeats needed to cause symptoms of Huntingtonās disease. Their work points to fewer than 185 CAGs as a threshold.
By Dr Chris Kay -
Youthful competitors: young brain cells oust the old
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!
-
Hunting for balance: how the huntingtin protein compensates in HD
Researchers look at the cause and effect of various forms of the HTT protein. They find both expanded and unexpanded HTT contribute to brain cell communication and the brain has an amazing capacity to compensate for changes related to disease
-
BAC to basics: a more accurate mouse model for Huntington's disease
A genetically-tweaked Huntington's disease mouse model shows a tendency for the CAG repeat to grow, just like we see in humans with the mutation.
-
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?
-
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.
-
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
-
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
-
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.