Silencing neuronal mutant androgen receptor in a mouse model of spinal and bulbar muscular atrophy

Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models

(2015) Yimin Hua et al.   GENES & DEVELOPMENT

Myopathic Involvement and Mitochondrial Pathology in Kennedy Disease and in Other Motor Neuron Diseases

(2014) D. Orsucci et al.   CURRENT MOLECULAR MEDICINE

Pioglitazone suppresses neuronal and muscular degeneration caused by polyglutamine-expanded androgen receptors

(2014) Madoka Iida et al.   HUMAN MOLECULAR GENETICS

Early onset and novel features in a spinal and bulbar muscular atrophy patient with a 68 CAG repeat

(2014) Christopher Grunseich et al.   NEUROMUSCULAR DISORDERS

Muscle Expression of Mutant Androgen Receptor Accounts for Systemic and Motor Neuron Disease Phenotypes in Spinal and Bulbar Muscular Atrophy

(2014) Constanza J. Cortes et al.   NEURON

Peripheral Androgen Receptor Gene Suppression Rescues Disease in Mouse Models of Spinal and Bulbar Muscular Atrophy

(2014) Andrew P. Lieberman et al.   Cell Reports

Degeneration of Neuromuscular Junction in Age and Dystrophy

(2014) Rüdiger Rudolf et al.   Frontiers in Aging Neuroscience

Pathological impact ofSMN2mis-splicing in adult SMA mice

(2013) Kentaro Sahashi et al.   EMBO Molecular Medicine

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

(2013) Timothy M Miller et al.   LANCET NEUROLOGY

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(2013) Carlo Rinaldi et al.   MOLECULAR MEDICINE

Heat shock factor-1 influences pathological lesion distribution of polyglutamine-induced neurodegeneration

(2013) Naohide Kondo et al.   Nature Communications

More than a bystander: the contributions of intrinsic skeletal muscle defects in motor neuron diseases

(2013) Justin G. Boyer et al.   Frontiers in Physiology

Neuronal Androgen Receptor Regulates Insulin Sensitivity via Suppression of Hypothalamic NF- B-Mediated PTP1B Expression

(2012) I.-C. Yu et al.   DIABETES

TSUNAMI: an antisense method to phenocopy splicing-associated diseases in animals

(2012) K. Sahashi et al.   GENES & DEVELOPMENT

Naratriptan mitigates CGRP1-associated motor neuron degeneration caused by an expanded polyglutamine repeat tract

(2012) Makoto Minamiyama et al.   NATURE MEDICINE

Genetic Biomarkers for ALS Disease in Transgenic SOD1G93A Mice

(2012) Ana C. Calvo et al.   PLoS One

Pathogenesis and therapy of spinal and bulbar muscular atrophy (SBMA)

(2012) Masahisa Katsuno et al.   PROGRESS IN NEUROBIOLOGY

Impaired motoneuronal retrograde transport in two models of SBMA implicates two sites of androgen action

(2011) Michael Q. Kemp et al.   HUMAN MOLECULAR GENETICS

Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model

(2011) Yimin Hua et al.   NATURE

Altered Expression of Myogenic Regulatory Factors in the Mouse Model of Amyotrophic Lateral Sclerosis

(2011) Raquel Manzano et al.   Neurodegenerative Diseases

Cell-autonomous and non-cell-autonomous toxicity in polyglutamine diseases

(2011) Fabio Sambataro et al.   PROGRESS IN NEUROBIOLOGY

Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

(2011) M. A. Passini et al.   Science Translational Medicine

RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

(2010) C. Frank Bennett et al.   Annual Review of Pharmacology and Toxicology

Myogenin and Class II HDACs Control Neurogenic Muscle Atrophy by Inducing E3 Ubiquitin Ligases

(2010) Viviana Moresi et al.   CELL

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

(2010) Y. Hua et al.   GENES & DEVELOPMENT

An Interdomain Interaction of the Androgen Receptor Is Required for Its Aggregation and Toxicity in Spinal and Bulbar Muscular Atrophy

(2010) Christopher R. Orr et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Disrupted Transforming Growth Factor-  Signaling in Spinal and Bulbar Muscular Atrophy

(2010) M. Katsuno et al.   JOURNAL OF NEUROSCIENCE

Native Functions of the Androgen Receptor Are Essential to Pathogenesis in a Drosophila Model of Spinobulbar Muscular Atrophy

(2010) Natalia B. Nedelsky et al.   NEURON

The profile of motor unit number estimation (MUNE) in spinal and bulbar muscular atrophy

(2009) K. Suzuki et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Conditional Inactivation of Androgen Receptor Gene in the Nervous System: Effects on Male Behavioral and Neuroendocrine Responses

(2009) K. Raskin et al.   JOURNAL OF NEUROSCIENCE

Standard and modified statistical mune evaluations in spinal-bulbar muscular atrophy

(2009) Tanya J. Lehky et al.   MUSCLE & NERVE

Mitochondrial abnormalities in spinal and bulbar muscular atrophy

(2008) Srikanth Ranganathan et al.   HUMAN MOLECULAR GENETICS

Spinal and bulbar muscular atrophy: Skeletal muscle pathology in male patients and heterozygous females

(2007) Gianni Sorarù et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES