Therapeutic strategies for spinal muscular atrophy: SMN and beyond

Therapeutic strategies for spinal muscular atrophy: SMN and beyond

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Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder characterized by loss of motor neurons and muscle atrophy, generally presenting in childhood. SMA is caused by low levels of the survival motor neuron protein (SMN) due to inactivating mutations in the encoding gene SMN1. A second...

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Xehetasun bibliografikoak
Egile Nagusiak:	Bowerman, M, Becker, C, Yáñez-Muñoz, R, Ning, K, Wood, M, Gillingwater, T, Talbot, K, UK SMA Research Consortium
Formatua:	Journal article
Argitaratua:	Company of Biologists 2017

Antzeko izenburuak

Therapeutic strategies for spinal muscular atrophy: SMN and beyond
nork: Bowerman, M, et al.
Argitaratua: (2017)

Therapeutic strategies for spinal muscular atrophy: SMN and beyond
nork: Melissa Bowerman, et al.
Argitaratua: (2017-08-01)

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nork: Winkelsas, AM, et al.
Argitaratua: (2021)

Targeting the 5′ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy
nork: Audrey M. Winkelsas, et al.
Argitaratua: (2021-03-01)

SMN deficiency disrupts brain development in a mouse model of severe spinal muscular atrophy.
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Argitaratua: (2010)

Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape
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Argitaratua: (2017)

SMN post-translational modifications in spinal muscular atrophy
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Argitaratua: (2023-02-01)

Spinal muscular atrophy in Venezuela: quantitative analysis of SMN1 and SMN2 genes
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Argitaratua: (2020-06-01)

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Argitaratua: (2011)

Therapy Development for Spinal Muscular Atrophy in SMN Independent Targets
nork: Li-Kai Tsai
Argitaratua: (2012-01-01)

CORRIGENDUM to Molecular Crosstalk Between Non-SMN-Related and SMN-Related Spinal Muscular Atrophy
Argitaratua: (2020-04-01)

The Need for SMN-Independent Treatments of Spinal Muscular Atrophy (SMA) to Complement SMN-Enhancing Drugs
nork: Niko Hensel, et al.
Argitaratua: (2020-02-01)

Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy
nork: Tejal Aslesh, et al.
Argitaratua: (2022-01-01)

SMN deficiency perturbs monoamine neurotransmitter metabolism in spinal muscular atrophy
nork: Valeria Valsecchi, et al.
Argitaratua: (2023-11-01)

The spinal muscular atrophy disease protein SMN is linked to the Golgi network.
nork: Chen-Hung Ting, et al.
Argitaratua: (2012-01-01)

In Vivo Translatome Profiling in Spinal Muscular Atrophy Reveals a Role for SMN Protein in Ribosome Biology
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Patient-specific responses to SMN2 splice-modifying treatments in spinal muscular atrophy fibroblasts
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Argitaratua: (2024-12-01)

Quantitative and structural features of the SMN1 and SMN2 genes in patients with spinal muscular atrophy 5q
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Argitaratua: (2024-12-01)

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Argitaratua: (2020)

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Argitaratua: (2001)

Targeting SR proteins improves SMN expression in spinal muscular atrophy cells.
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Argitaratua: (2014-01-01)

The contribution of mouse models to understanding the pathogenesis of spinal muscular atrophy
nork: James N. Sleigh, et al.
Argitaratua: (2011-07-01)

Molecular and Epidemiological Characterization of SMN Genes in Cuban Patients with Spinal Muscular Atrophy
nork: Fabián Lombillo-Alfonso, et al.
Argitaratua: (2020-01-01)

Molecular And Epidemiological Characterization Of SMN Genes In Cuban Patients With Spinal Muscular Atrophy
nork: Fabián Lombillo, et al.
Argitaratua: (2019-05-01)

What could be the function of the spinal muscular atrophy-causing protein SMN in macrophages?
nork: Ines Tapken, et al.
Argitaratua: (2024-05-01)

Development and Translation of Therapies for Spinal Muscular Atrophy
nork: Hannah K. Shorrock, et al.
Argitaratua: (2016-07-01)

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nork: Murray, L, et al.
Argitaratua: (2010)

Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy
nork: Winkelsas, AM
Argitaratua: (2020)

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nork: Matthew E.R. Butchbach, et al.
Argitaratua: (2007-08-01)

A Single Amino Acid Residue Regulates PTEN-Binding and Stability of the Spinal Muscular Atrophy Protein SMN
nork: Sebastian Rademacher, et al.
Argitaratua: (2020-11-01)

The neuromuscular impact of symptomatic SMN restoration in a mouse model of spinal muscular atrophy
nork: W. Arnold, et al.
Argitaratua: (2016-03-01)

Gene therapy for spinal muscular atrophy: perspectives on the possibility of optimizing SMN1 delivery to correct all neurological and systemic perturbations
nork: Sharon J. Brown, et al.
Argitaratua: (2025-07-01)

Stability and Oligomerization of Mutated SMN Protein Determine Clinical Severity of Spinal Muscular Atrophy
nork: Niba, Emma Tabe Eko, et al.
Argitaratua: (2022)

Stability and Oligomerization of Mutated SMN Protein Determine Clinical Severity of Spinal Muscular Atrophy
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Argitaratua: (2022)

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Argitaratua: (2024-01-01)

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nork: Anderton Ryan, et al.
Argitaratua: (2014-03-01)

Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes
nork: Amparo Garcia-Lopez, et al.
Argitaratua: (2018-05-01)

Analysis of the survival of motor neuron (SMN1) gene mutations in Spinal Muscular Atrophy through models
nork: Owen, N, et al.
Argitaratua: (2002)

Chapter 16 Spinal Muscular Atrophy
nork: Talbot, K, et al.
Argitaratua: (2003)