Combined treatment with the histone deacetylase inhibitor LBH589 and a splice‐switch antisense oligonucleotide enhances SMN2 splicing and SMN expression in Spinal Muscular Atrophy cells
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Title
Combined treatment with the histone deacetylase inhibitor LBH589 and a splice‐switch antisense oligonucleotide enhances SMN2 splicing and SMN expression in Spinal Muscular Atrophy cells
Authors
Keywords
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Journal
JOURNAL OF NEUROCHEMISTRY
Volume -, Issue -, Pages -
Publisher
Wiley
Online
2019-12-07
DOI
10.1111/jnc.14935
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Note: Only part of the references are listed.- Two breakthrough gene-targeted treatments for spinal muscular atrophy: challenges remain
- (2018) Charlotte J. Sumner et al. JOURNAL OF CLINICAL INVESTIGATION
- Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
- (2018) Eugenio Mercuri et al. NEW ENGLAND JOURNAL OF MEDICINE
- Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)
- (2018) Hasane Ratni et al. JOURNAL OF MEDICINAL CHEMISTRY
- Clinical trial of L-Carnitine and valproic acid in spinal muscular atrophy type I
- (2017) Kristin J. Krosschell et al. MUSCLE & NERVE
- Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
- (2017) Jerry R. Mendell et al. NEW ENGLAND JOURNAL OF MEDICINE
- Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
- (2017) Richard S. Finkel et al. NEW ENGLAND JOURNAL OF MEDICINE
- Transcript, methylation and molecular docking analyses of the effects of HDAC inhibitors, SAHA and Dacinostat, on SMN2 expression in fibroblasts of SMA patients
- (2016) Jafar Mohseni et al. JOURNAL OF HUMAN GENETICS
- Sam68 promotes self-renewal and glycolytic metabolism in mouse neural progenitor cells by modulating Aldh1a3 pre-mRNA 3'-end processing
- (2016) Piergiorgio La Rosa et al. eLife
- Developing therapies for spinal muscular atrophy
- (2015) Mary H. Wertz et al. Annals of the New York Academy of Sciences
- SAM68 is a physiological regulator ofSMN2splicing in spinal muscular atrophy
- (2015) Vittoria Pagliarini et al. JOURNAL OF CELL BIOLOGY
- Spinal Muscular Atrophy: From Gene Discovery to Clinical Trials
- (2013) Dian K. Nurputra et al. ANNALS OF HUMAN GENETICS
- Spinal muscular atrophy: Development and implementation of potential treatments
- (2013) W. David Arnold et al. ANNALS OF NEUROLOGY
- Histone deacetylase inhibitors as potential treatment for spinal muscular atrophy
- (2013) Jafar Mohseni et al. GENETICS AND MOLECULAR BIOLOGY
- SMA valiant trial: A prospective, double-blind, placebo-controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy
- (2013) John T. Kissel et al. MUSCLE & NERVE
- Increasing SMN levels using the histone deacetylase inhibitor SAHA ameliorates defects in skeletal muscle microvasculature in a mouse model of severe spinal muscular atrophy
- (2013) Eilidh Somers et al. NEUROSCIENCE LETTERS
- Valproic acid increases SMN2 expression and modulates SF2/ASF and hnRNPA1 expression in SMA fibroblast cell lines
- (2011) Indra Sari Kusuma Harahap et al. BRAIN & DEVELOPMENT
- Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model
- (2011) Yimin Hua et al. NATURE
- The splicing regulator Sam68 binds to a novel exonic splicing silencer and functions in SMN2 alternative splicing in spinal muscular atrophy
- (2010) Simona Pedrotti et al. EMBO JOURNAL
- A degron created by SMN2 exon 7 skipping is a principal contributor to spinal muscular atrophy severity
- (2010) S. Cho et al. GENES & DEVELOPMENT
- LBH589 induces up to 10-fold SMN protein levels by several independent mechanisms and is effective even in cells from SMA patients non-responsive to valproate
- (2009) Lutz Garbes et al. HUMAN MOLECULAR GENETICS
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