Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

Autor:	Robert C. Bucelli, Manjit McNeill, Shafeeq Ladha, Angela Genge, Stephanie Fradette, Ivan Nestorov, John Ravits, Philip Van Damme, Roger Lane, Christopher J McDermott, Jonathan Glass, Merit Cudkowicz, Laura Fanning, C. Frank Bennett, Heiko Runz, Ih Chang, Hani Houshyar, Toby A. Ferguson, Alfred Sandrock, Alan Pestronk, Danielle Graham, Timothy M. Miller, Randall G. Trudell, Nazem Atassi, Peter M. Andersen, François Salachas, Nicholas J. Maragakis, Albert L. Ludolph, Pamela J. Shaw, Lorne Zinman, Alexander McCampbell
Rok vydání:	2020
Předmět:	Messenger RNA Mutation biology business.industry Oligonucleotide animal diseases SOD1 nutritional and metabolic diseases General Medicine 030204 cardiovascular system & hematology Intrathecal medicine.disease_cause Molecular biology nervous system diseases Superoxide dismutase 03 medical and health sciences 0302 clinical medicine nervous system Antisense oligonucleotides Protein biosynthesis biology.protein Medicine 030212 general & internal medicine business
Zdroj:	New England Journal of Medicine. 383:109-119
ISSN:	1533-4406 0028-4793
Popis:	Background Tofersen is an antisense oligonucleotide that mediates the degradation of superoxide dismutase 1 (SOD1) messenger RNA to reduce SOD1 protein synthesis. Intrathecal administratio...
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_________::cd89d37b574703a54dc8fa55df6264a4 https://doi.org/10.1056/nejmoa2003715 Zobrazit plný text záznamu