John Ravits

Amyotrophic lateral sclerosis caused by hexanucleotide repeat expansions in C9orf72: from genetics to therapeutics

Neuronal aging causes mislocalization of splicing proteins and unchecked cellular stress

TDP-43 loss induces cryptic polyadenylation in ALS/FTD

Autoimmune response to C9orf72 protein in amyotrophic lateral sclerosis

RNA triggers chronic stress during neuronal aging

Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study

CK1δ/ε kinases regulate TDP-43 phosphorylation and are therapeutic targets for ALS-related TDP-43 hyperphosphorylation

Author Correction: TDP-43 loss and ALS-risk SNPs drive mis-splicing and depletion of UNC13A

Alternatively spliced ELAVL3 cryptic exon 4a causes ELAVL3 downregulation in ALS TDP-43 proteinopathy.

Stathmin-2 loss leads to neurofilament-dependent axonal collapse driving motor and sensory denervation

Challenges of profiling motor neuron transcriptomes from human spinal cord

TDP-43 loss and ALS-risk SNPs drive mis-splicing and depletion of UNC13A

Heat-shock chaperone HSPB1 regulates cytoplasmic TDP-43 phase separation and liquid-to-gel transition

Nuclear RIPK1 promotes chromatin remodeling to mediate inflammatory response

Cell environment shapes TDP-43 function with implications in neuronal and muscle disease

Enrichment of SARM1 alleles encoding variants with constitutively hyperactive NADase in patients with ALS and other motor nerve disorders

TDP-43 mediates SREBF2-regulated gene expression required for oligodendrocyte myelination

An integrated multi-omic analysis of iPSC-derived motor neurons from C9ORF72 ALS patients

Novel STMN2 Variant Linked to Amyotrophic Lateral Sclerosis Risk and Clinical Phenotype

Nuclear depletion of RNA-binding protein ELAVL3 (HuC) in sporadic and familial amyotrophic lateral sclerosis.

Heat shock chaperone HSPB1 regulates cytoplasmic TDP-43 phase separation and liquid-to-gel transition

Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for <i>SOD1</i> ALS

Primary lateral sclerosis: consensus diagnostic criteria

Reduced C9ORF72 function exacerbates gain of toxicity from ALS/FTD-causing repeat expansion in C9orf72

Validation of serum neurofilaments as prognostic and potential pharmacodynamic biomarkers for ALS

ALS motor neurons exhibit hallmark metabolic defects that are rescued by SIRT3 activation

Machine learning suggests polygenic risk for cognitive dysfunction in amyotrophic lateral sclerosis

Preface: promoting research in PLS: current knowledge and future challenges

Single-cell RNA-seq analysis of human iPSC-derived motor neurons resolves early and predictive ALS signatures

Premature polyadenylation-mediated loss of stathmin-2 is a hallmark of TDP-43-dependent neurodegeneration