The New England journal of medicine, 2019-06, Vol.380 (24), p.2307-2316
- 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
2019
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- Autor(en) / Beteiligte
- 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
- Titel
- Targeting Huntingtin Expression in Patients with Huntington’s Disease
- Ist Teil von
- The New England journal of medicine, 2019-06, Vol.380 (24), p.2307-2316
- Ort / Verlag
- United States: Massachusetts Medical Society
- Erscheinungsjahr
- 2019
- Quelle
- EZB-FREE-00999 freely available EZB journals
- Beschreibungen/Notizen
- 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 the drug.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0028-4793eISSN: 1533-4406DOI: 10.1056/NEJMoa1900907Titel-ID: cdi_proquest_journals_2239265965
- Format
- –
- Schlagworte
- Adult, Antisense oligonucleotides, Cerebrospinal fluid, Dose-Response Relationship, Drug, Drug dosages, Female, Humans, Huntingtin, Huntingtin Protein - antagonists & inhibitors, Huntingtin Protein - cerebrospinal fluid, Huntingtin Protein - genetics, Huntington Disease - drug therapy, Huntingtons disease, Injections, Spinal, Male, Middle Aged, mRNA, Mutation, Nervous system, Neurodegenerative diseases, Nucleotides - chemical synthesis, Nucleotides - pharmacology, Oligonucleotides - cerebrospinal fluid, Oligonucleotides - therapeutic use, Pharmaceuticals, Pharmacokinetics, Polyglutamine, Proteins, Trinucleotide repeats