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
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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

Prof Josef Priller

Group Leader

Defining and modulating myeloid cell function in neurodegenerative diseases

Prof Josef Priller