Raise the Roof: Boosting the Efficacy of a Spinal Muscular Atrophy Therapy
Author:
Publisher
Elsevier BV
Subject
General Neuroscience
Reference10 articles.
1. The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy
2. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
3. The SMN complex
4. Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model
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1. The development and controversy of competitive endogenous RNA hypothesis in non-coding genes;Molecular and Cellular Biochemistry;2020-09-25
2. Diagnostische Verfahren;Diagnostik und Therapie neurologischer Erkrankungen;2020
3. Spinal Muscular Atrophy Modeling and Treatment Advances by Induced Pluripotent Stem Cells Studies;Stem Cell Reviews and Reports;2019-08-07
4. Small-molecule flunarizine increases SMN protein in nuclear Cajal bodies and motor function in a mouse model of spinal muscular atrophy;Scientific Reports;2018-02-01
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