4.5 Review

The SMA Trust: the role of a disease-focused research charity in developing treatments for SMA

Related references

Note: Only part of the references are listed.
Article Anatomy & Morphology

Survival of motor neurone protein is required for normal postnatal development of the spleen

Alison K. Thomson et al.

JOURNAL OF ANATOMY (2017)

Article Genetics & Heredity

The Power of Human Protective Modifiers: PLS3 and CORO1C Unravel Impaired Endocytosis in Spinal Muscular Atrophy and Rescue SMA Phenotype

Seyyedmohsen Hosseinibarkooie et al.

AMERICAN JOURNAL OF HUMAN GENETICS (2016)

Article Clinical Neurology

Vascular Defects and Spinal Cord Hypoxia in Spinal Muscular Atrophy

Eilidh Somers et al.

ANNALS OF NEUROLOGY (2016)

Article Multidisciplinary Sciences

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

Suzan M. Hammond et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2016)

Article Multidisciplinary Sciences

Survival Motor Neuron (SMN) protein is required for normal mouse liver development

Eva Szunyogova et al.

SCIENTIFIC REPORTS (2016)

Article Medicine, Research & Experimental

Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy

Rachael A. Powis et al.

JCI INSIGHT (2016)

Article Clinical Neurology

A Large Animal Model of Spinal Muscular Atrophy and Correction of Phenotype

Sandra I. Duque et al.

ANNALS OF NEUROLOGY (2015)

Editorial Material Pharmacology & Pharmacy

Moving towards treatments for spinal muscular atrophy: hopes and limits

Brunhilde Wirth et al.

EXPERT OPINION ON EMERGING DRUGS (2015)

Article Biotechnology & Applied Microbiology

PTEN Depletion Decreases Disease Severity and Modestly Prolongs Survival in a Mouse Model of Spinal Muscular Atrophy

Daniel Little et al.

MOLECULAR THERAPY (2015)

Article Neurosciences

Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice

Yannick Tanguy et al.

FRONTIERS IN MOLECULAR NEUROSCIENCE (2015)

Article Biochemistry & Molecular Biology

Chondrolectin affects cell survival and neuronal outgrowth in in vitro and in vivo models of spinal muscular atrophy

James N. Sleigh et al.

HUMAN MOLECULAR GENETICS (2014)

Article Medicine, Research & Experimental

Dysregulation of ubiquitin homeostasis and β-catenin signaling promote spinal muscular atrophy

Thomas M. Wishart et al.

JOURNAL OF CLINICAL INVESTIGATION (2014)

Article Medicine, Research & Experimental

Requirement of enhanced Survival Motoneuron protein imposed during neuromuscular junction maturation

Shingo Kariya et al.

JOURNAL OF CLINICAL INVESTIGATION (2014)

Article Biotechnology & Applied Microbiology

Intramuscular scAAV9-SMN Injection Mediates Widespread Gene Delivery to the Spinal Cord and Decreases Disease Severity in SMA Mice

Sofia Benkhelifa-Ziyyat et al.

MOLECULAR THERAPY (2013)

Review Biochemistry & Molecular Biology

Spinal muscular atrophy: going beyond the motor neuron

Gillian Hamilton et al.

TRENDS IN MOLECULAR MEDICINE (2013)

Review Cell Biology

The contribution of mouse models to understanding the pathogenesis of spinal muscular atrophy

James N. Sleigh et al.

DISEASE MODELS & MECHANISMS (2011)

Article Biochemistry & Molecular Biology

Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

Elisa Dominguez et al.

HUMAN MOLECULAR GENETICS (2011)

Article Medicine, Research & Experimental

Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy

Cathleen M. Lutz et al.

JOURNAL OF CLINICAL INVESTIGATION (2011)

Article Biochemistry & Molecular Biology

PTEN depletion rescues axonal growth defect and improves survival in SMN-deficient motor neurons

Ke Ning et al.

HUMAN MOLECULAR GENETICS (2010)

Article Cell Biology

Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular Atrophy

Chiara F. Valori et al.

SCIENCE TRANSLATIONAL MEDICINE (2010)

Review Neurosciences

Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?

Arthur H. M. Burghes et al.

NATURE REVIEWS NEUROSCIENCE (2009)