Therapeutic strategies for spinal muscular atrophy: SMN and beyond

Therapeutic strategies for spinal muscular atrophy: SMN and beyond

عرض إصدارات أخرى (1)

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...

وصف كامل

التفاصيل البيبلوغرافية
المؤلفون الرئيسيون:	Bowerman, M, Becker, C, Yáñez-Muñoz, R, Ning, K, Wood, M, Gillingwater, T, Talbot, K, UK SMA Research Consortium
التنسيق:	Journal article
منشور في:	Company of Biologists 2017

مواد مشابهة

Therapeutic strategies for spinal muscular atrophy: SMN and beyond
حسب: Bowerman, M, وآخرون
منشور في: (2017)

Therapeutic strategies for spinal muscular atrophy: SMN and beyond
حسب: Melissa Bowerman, وآخرون
منشور في: (2017-08-01)

Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy
حسب: Winkelsas, AM, وآخرون
منشور في: (2021)

Targeting the 5′ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy
حسب: Audrey M. Winkelsas, وآخرون
منشور في: (2021-03-01)

SMN deficiency disrupts brain development in a mouse model of severe spinal muscular atrophy.
حسب: Wishart, T, وآخرون
منشور في: (2010)

Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape
حسب: Wood, M, وآخرون
منشور في: (2017)

SMN post-translational modifications in spinal muscular atrophy
حسب: Giulietta M. Riboldi, وآخرون
منشور في: (2023-02-01)

Spinal muscular atrophy in Venezuela: quantitative analysis of SMN1 and SMN2 genes
حسب: Yuri Yépez, وآخرون
منشور في: (2020-06-01)

Molecular Crosstalk Between Non-SMN-Related and SMN-Related Spinal Muscular Atrophy
حسب: Darija Šoltić, وآخرون
منشور في: (2020-03-01)

The contribution of mouse models to understanding the pathogenesis of spinal muscular atrophy.
حسب: Sleigh, J, وآخرون
منشور في: (2011)

Therapy Development for Spinal Muscular Atrophy in SMN Independent Targets
حسب: Li-Kai Tsai
منشور في: (2012-01-01)

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

The Need for SMN-Independent Treatments of Spinal Muscular Atrophy (SMA) to Complement SMN-Enhancing Drugs
حسب: Niko Hensel, وآخرون
منشور في: (2020-02-01)

Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy
حسب: Tejal Aslesh, وآخرون
منشور في: (2022-01-01)

SMN deficiency perturbs monoamine neurotransmitter metabolism in spinal muscular atrophy
حسب: Valeria Valsecchi, وآخرون
منشور في: (2023-11-01)

The spinal muscular atrophy disease protein SMN is linked to the Golgi network.
حسب: Chen-Hung Ting, وآخرون
منشور في: (2012-01-01)

In Vivo Translatome Profiling in Spinal Muscular Atrophy Reveals a Role for SMN Protein in Ribosome Biology
حسب: Paola Bernabò, وآخرون
منشور في: (2017-10-01)

Patient-specific responses to SMN2 splice-modifying treatments in spinal muscular atrophy fibroblasts
حسب: Ilaria Signoria, وآخرون
منشور في: (2024-12-01)

Quantitative and structural features of the SMN1 and SMN2 genes in patients with spinal muscular atrophy 5q
حسب: A. I. Vlasenko, وآخرون
منشور في: (2024-12-01)

A single amino acid residue regulates PTEN-binding and stability of the spinal muscular atrophy protein SMN
حسب: Rademacher, S, وآخرون
منشور في: (2020)

Spinal muscular atrophy.
حسب: Talbot, K, وآخرون
منشور في: (2001)

Targeting SR proteins improves SMN expression in spinal muscular atrophy cells.
حسب: Claribel D Wee, وآخرون
منشور في: (2014-01-01)

The contribution of mouse models to understanding the pathogenesis of spinal muscular atrophy
حسب: James N. Sleigh, وآخرون
منشور في: (2011-07-01)

Molecular and Epidemiological Characterization of SMN Genes in Cuban Patients with Spinal Muscular Atrophy
حسب: Fabián Lombillo-Alfonso, وآخرون
منشور في: (2020-01-01)

Molecular And Epidemiological Characterization Of SMN Genes In Cuban Patients With Spinal Muscular Atrophy
حسب: Fabián Lombillo, وآخرون
منشور في: (2019-05-01)

What could be the function of the spinal muscular atrophy-causing protein SMN in macrophages?
حسب: Ines Tapken, وآخرون
منشور في: (2024-05-01)

Development and Translation of Therapies for Spinal Muscular Atrophy
حسب: Hannah K. Shorrock, وآخرون
منشور في: (2016-07-01)

Review: neuromuscular synaptic vulnerability in motor neurone disease: amyotrophic lateral sclerosis and spinal muscular atrophy.
حسب: Murray, L, وآخرون
منشور في: (2010)

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

Abnormal motor phenotype in the SMNΔ7 mouse model of spinal muscular atrophy
حسب: Matthew E.R. Butchbach, وآخرون
منشور في: (2007-08-01)

A Single Amino Acid Residue Regulates PTEN-Binding and Stability of the Spinal Muscular Atrophy Protein SMN
حسب: Sebastian Rademacher, وآخرون
منشور في: (2020-11-01)

The neuromuscular impact of symptomatic SMN restoration in a mouse model of spinal muscular atrophy
حسب: W. Arnold, وآخرون
منشور في: (2016-03-01)

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

Stability and Oligomerization of Mutated SMN Protein Determine Clinical Severity of Spinal Muscular Atrophy
حسب: Niba, Emma Tabe Eko, وآخرون
منشور في: (2022)

Stability and Oligomerization of Mutated SMN Protein Determine Clinical Severity of Spinal Muscular Atrophy
حسب: Niba, Emma Tabe Eko, وآخرون
منشور في: (2022)

P719: SMN1 deletion and silent carrier screening for spinal muscular atrophy
حسب: Poh San Lai, وآخرون
منشور في: (2024-01-01)

Investigation of a recombinant SMN protein delivery system to treat spinal muscular atrophy
حسب: Anderton Ryan, وآخرون
منشور في: (2014-03-01)

Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes
حسب: Amparo Garcia-Lopez, وآخرون
منشور في: (2018-05-01)

Analysis of the survival of motor neuron (SMN1) gene mutations in Spinal Muscular Atrophy through models
حسب: Owen, N, وآخرون
منشور في: (2002)

Chapter 16 Spinal Muscular Atrophy
حسب: Talbot, K, وآخرون
منشور في: (2003)