Preservation of forelimb function by UPF1 gene therapy in a rat model of TDP-43-induced motor paralysis

A post-translational regulatory switch on UPF1 controls targeted mRNA degradation

(2014) Tatsuaki Kurosaki et al.   GENES & DEVELOPMENT

Organizing Principles of Mammalian Nonsense-Mediated mRNA Decay

(2013) Maximilian Wei-Lin Popp et al.   Annual Review of Genetics

Regulation of nonsense-mediated mRNA decay: Implications for physiology and disease

(2013) Rachid Karam et al.   Biochimica et Biophysica Acta-Gene Regulatory Mechanisms

Inhibition of Nonsense-Mediated RNA Decay Activates Autophagy

(2013) J. Wengrod et al.   MOLECULAR AND CELLULAR BIOLOGY

Selective Forelimb Impairment in Rats Expressing a Pathological TDP-43 25 kDa C-terminal Fragment to Mimic Amyotrophic Lateral Sclerosis

(2013) Robert D Dayton et al.   MOLECULAR THERAPY

Therapeutic modulation of eIF2α phosphorylation rescues TDP-43 toxicity in amyotrophic lateral sclerosis disease models

(2013) Hyung-Jun Kim et al.   NATURE GENETICS

Rules that govern UPF1 binding to mRNA 3′ UTRs

(2013) Tatsuaki Kurosaki et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The advent of AAV9 expands applications for brain and spinal cord gene delivery

(2012) Robert D Dayton et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Autoregulation of TDP-43 mRNA levels involves interplay between transcription, splicing, and alternative polyA site selection

(2012) S. E. Avendano-Vazquez et al.   GENES & DEVELOPMENT

Inhibition of RNA lariat debranching enzyme suppresses TDP-43 toxicity in ALS disease models

(2012) Maria Armakola et al.   NATURE GENETICS

NMD: a multifaceted response to premature translational termination

(2012) Stephanie Kervestin et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Adeno-associated virus (AAV) gene therapy for neurological disease

(2012) Marc S. Weinberg et al.   NEUROPHARMACOLOGY

Methylene Blue Protects against TDP-43 and FUS Neuronal Toxicity in C. elegans and D. rerio

(2012) Alexandra Vaccaro et al.   PLoS One

Autophagy activators rescue and alleviate pathogenesis of a mouse model with proteinopathies of the TAR DNA-binding protein 43

(2012) I.-F. Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Genetic strategies to study TDP-43 in rodents and to develop preclinical therapeutics for amyotrophic lateral sclerosis

(2011) David B. Wang et al.   EUROPEAN JOURNAL OF NEUROSCIENCE

Correction of Neurological Disease of Mucopolysaccharidosis IIIB in Adult Mice by rAAV9 Trans-Blood–Brain Barrier Gene Delivery

(2011) Haiyan Fu et al.   MOLECULAR THERAPY

Long pre-mRNA depletion and RNA missplicing contribute to neuronal vulnerability from loss of TDP-43

(2011) Magdalini Polymenidou et al.   NATURE NEUROSCIENCE

A Yeast Model of FUS/TLS-Dependent Cytotoxicity

(2011) Shulin Ju et al.   PLOS BIOLOGY

TDP-43 regulates its mRNA levels through a negative feedback loop

(2010) Youhna M Ayala et al.   EMBO JOURNAL

Cytoplasmic Mislocalization of TDP-43 Is Toxic to Neurons and Enhanced by a Mutation Associated with Familial Amyotrophic Lateral Sclerosis

(2010) S. J. Barmada et al.   JOURNAL OF NEUROSCIENCE

Expansive Gene Transfer in the Rat CNS Rapidly Produces Amyotrophic Lateral Sclerosis Relevant Sequelae When TDP-43 is Overexpressed

(2010) David B Wang et al.   MOLECULAR THERAPY

TAR DNA-binding protein 43 in neurodegenerative disease

(2010) Alice S. Chen-Plotkin et al.   Nature Reviews Neurology

TDP-43 transgenic mice develop spastic paralysis and neuronal inclusions characteristic of ALS and frontotemporal lobar degeneration

(2010) Hans Wils et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

TARDBP 3′-UTR variant in autopsy-confirmed frontotemporal lobar degeneration with TDP-43 proteinopathy

(2009) Michael A. Gitcho et al.   ACTA NEUROPATHOLOGICA

Mimicking Aspects of Frontotemporal Lobar Degeneration and Lou Gehrig's Disease in Rats via TDP-43 Overexpression

(2009) Jason B Tatom et al.   MOLECULAR THERAPY

Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons

(2009) Sandra Duque et al.   MOLECULAR THERAPY

Upf1 Phosphorylation Triggers Translational Repression during Nonsense-Mediated mRNA Decay

(2008) Olaf Isken et al.   CELL

SMD and NMD are competitive pathways that contribute to myogenesis: effects on PAX3 and myogenin mRNAs

(2008) C. Gong et al.   GENES & DEVELOPMENT

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY