N Engl J Med
Published

Targeting Huntingtin Expression in Patients with Huntington's Disease.

Authors

Sarah J Tabrizi, Blair R Leavitt, G Bernhard Landwehrmeyer, Edward J Wild, Carsten Saft, Roger A Barker, Nick F Blair, David Craufurd, Josef Priller, Hugh Rickards, Anne Rosser, Holly B Kordasiewicz, Christian Czech, Eric E Swayze, Daniel A Norris, Tiffany Baumann, Irene Gerlach, Scott A Schobel, Erika Paz, Anne V Smith, C Frank Bennett, Roger M Lane,
Read More

Abstract

Huntington's disease is an autosomal-dominant neurodegenerative disease caused by CAG trinucleotide repeat expansion in HTT, resulting in a mutant huntingtin protein. IONIS-HTTRx (hereafter, HTTRx) is an antisense oligonucleotide designed to inhibit HTT messenger RNA and thereby reduce concentrations of mutant huntingtin.

PMID:31059641 | DOI:

UK DRI Authors

Josef Priller

Prof Josef Priller

Group Leader

Defining and modulating myeloid cell function in neurodegenerative diseases

Prof Josef Priller
Paul Matthews

Prof Paul Matthews

Group Leader

Exploring neuronal vulnerability and genetic risk variants in Alzheimer’s progression

Prof Paul Matthews