Articles with the topic: gene-silencing

Exciting new Huntingtin lowering tool described

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.

Dr Jeff CarrollAugust 05, 2019

Details emerge of first Huntington’s disease gene therapy clinical trial

Details emerge of first Huntington’s 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 ‘single-shot’ virus delivery system.

Dr Anna PfalzerJuly 17, 2019

Fresh Updates from First Huntingtin Lowering Study Publication

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

Dr Jeff CarrollMay 07, 2019

Huntington's disease goes viral as UniQure inches ahead in gene therapy race

Huntington's disease goes viral as UniQure inches ahead in gene therapy race

FDA grants “Investigational New Drug” status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntington’s Disease patients

Dr Jeff CarrollJanuary 30, 2019

Advances on many fronts in the battle against the protein that causes Huntington's disease

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

Dr Jeff CarrollDecember 04, 2018

HDSA FAQ on the Roche/Genentech RG6042 program

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)

Leora FoxOctober 19, 2018

Roche announces details of its 'pivotal' huntingtin-lowering study

Roche announces details of its 'pivotal' huntingtin-lowering study

The GENERATION-HD1 trial will test whether RG6042 – formerly Ionis-HTTRx – slows the progression of Huntington's disease

Dr Jeff CarrollSeptember 17, 2018

HDSA’s Q&A on the latest huntingtin-lowering update from Ionis and Roche

HDSA’s Q&A on the latest huntingtin-lowering update from Ionis and Roche

Our friends at @hdsa have produced this really good Q&A document about the latest huntingtin-lowering news.

Leora FoxApril 26, 2018

Huntington's disease therapeutics conference 2018 - day 3

Huntington's disease therapeutics conference 2018 - day 3

Updates from day 3 of the Huntington’s Disease Therapeutics Conference: huntingtin protein - and lowering it

Dr Jeff CarrollMarch 02, 2018