gene-silencing
-
Stopping C-A-G Repeat Expansion In Its Tracks
A new study shows that lowering MSH3, a key DNA repair protein, with antisense oligonucleotide (ASO) therapy can stop CAG repeat expansions in HD patient-derived brain cells. This could be a promising way to slow symptom onset and progression.
-
Sad News from Roche and Ionis – ASO Trial Halted Early
Disappointing news from Roche and Ionis; the phase III Tominersen huntingtin-lowering trial has been halted early
-
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
-
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.
-
Details emerge of first Huntingtonâs disease gene therapy clinical trial
UniQure announces key details of its planned trial to assess the safety and ability of AMT-130 gene therapy to lower the problematic huntingtin protein using a âsingle-shotâ virus delivery system.
-
Fresh Updates from First Huntingtin Lowering Study Publication
Hot off the presses – New publication gives more details about the results of Ionis and Roche's safety study with a Huntingtin-lowering ASO
-
Huntington's disease goes viral as UniQure inches ahead in gene therapy race
FDA grants âInvestigational New Drugâ status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntingtonâs Disease patients
-
Advances on many fronts in the battle against the protein that causes Huntington's disease
This fall sees exciting announcements from a number of companies focused on novel Huntingtin Lowering technologies, including Wave, PTC and Voyager
-
HDSA FAQ on the Roche/Genentech RG6042 program
HDSA's frequently asked questions factsheet about the huntingtin-lowering trial ASO program was so good we stole it (with permission)
By Dr Leora Fox -
Roche announces details of its 'pivotal' huntingtin-lowering study
The GENERATION-HD1 trial will test whether RG6042 â formerly Ionis-HTTRx â slows the progression of Huntington's disease