Targeting Huntingtin Expression in Patients with Huntington’s Disease
Targeting Huntingtin Expression in Patients with Huntington’s Disease
About this item
Full title
Author / Creator
Tabrizi, Sarah J , Leavitt, Blair R , Landwehrmeyer, G. Bernhard , Wild, Edward J , Saft, Carsten , Barker, Roger A , Blair, Nick F , Craufurd, David , Priller, Josef , Rickards, Hugh , Rosser, Anne , Kordasiewicz, Holly B , Czech, Christian , Swayze, Eric E , Norris, Daniel A , Baumann, Tiffany , Gerlach, Irene , Schobel, Scott A , Paz, Erika , Smith, Anne V , Bennett, C. Frank , Lane, Roger M and Phase 1–2a IONIS-HTTRx Study Site Teams
Publisher
United States: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
United States: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
Mutated
HTT
, resulting in mutant huntingtin, causes Huntington’s disease. A phase 1–2a trial of intrathecal delivery of an antisense oligonucleotide targeting
HTT
mRNA in 34 persons with Huntington’s disease showed a dose-dependent reduction of mutant huntingtin in cerebrospinal fluid and no serious adverse events in those who received...
Alternative Titles
Full title
Targeting Huntingtin Expression in Patients with Huntington’s Disease
Authors, Artists and Contributors
Author / Creator
Leavitt, Blair R
Landwehrmeyer, G. Bernhard
Wild, Edward J
Saft, Carsten
Barker, Roger A
Blair, Nick F
Craufurd, David
Priller, Josef
Rickards, Hugh
Rosser, Anne
Kordasiewicz, Holly B
Czech, Christian
Swayze, Eric E
Norris, Daniel A
Baumann, Tiffany
Gerlach, Irene
Schobel, Scott A
Paz, Erika
Smith, Anne V
Bennett, C. Frank
Lane, Roger M
Phase 1–2a IONIS-HTTRx Study Site Teams
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_journals_2239265965
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_2239265965
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa1900907
