A new imaging tool means that scientists can now directly measure the levels of the toxic huntingtin protein in animal models of Huntington’s disease, letting us see how well huntingtin lowering therapies are working in their brains
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.
Success in Huntington’s disease cells and mice for zinc finger drugs that reduce production of the harmful protein
Scientists make precise changes to the DNA of a live animal. Could it work for human genetic diseases?