Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial-Reference-Cited by-同舟云学术

Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial

Published:2023-03 Issue:3 Volume:22 Page:218-228
ISSN:1474-4422
Container-title:The Lancet Neurology
language:en
Short-container-title:The Lancet Neurology

Author:

Thornton Charles A,Moxley Richard Thomas,Eichinger Katy,Heatwole Chad,Mignon Laurence,Arnold W David,Ashizawa Tetsuo,Day John W,Dent Gersham,Tanner Matthew K,Duong Tina,Greene Ericka P,Herbelin Laura,Johnson Nicholas E,King Wendy,Kissel John T,Leung Doris G,Lott Donovan J,Norris Daniel A,Pucillo Evan M,Schell Wendy,Statland Jeffrey M,Stinson Nikia,Subramony Sub H,Xia Shuting,Bishop Kathie M,Bennett C Frank

Publisher

Elsevier BV

Subject

Neurology (clinical)

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3. RNA-binding proteins in microsatellite expansion disorders: mediators of RNA toxicity;Echeverria;Brain Res,2012

4. Myotonic dystrophy type 1 is associated with nuclear foci of mutant RNA, sequestration of muscleblind proteins and deregulated alternative splicing in neurons;Jiang;Hum Mol Genet,2004

5. Recruitment of human muscleblind proteins to (CUG)(n) expansions associated with myotonic dystrophy;Miller;EMBO J,2000

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