Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72-associated amyotrophic lateral sclerosis
Frontiers | C9ORF72: What It Is, What It Does, and Why It Matters | Cellular Neuroscience
Biogen and Ionis Announce Topline Phase 1 Study Results of Investigational Drug in C9orf72 Amyotrophic Lateral Sclerosis | Biogen
C9orf72-mediated ALS and FTD: multiple pathways to disease | Nature Reviews Neurology
Knockdown of C9ORF72 with ASOs suppresses RNA foci in ALS motor... | Download Scientific Diagram
RNA-mediated toxicity in C9orf72 ALS and FTD - ScienceDirect
IJMS | Free Full-Text | Gene Therapy for ALS—A Perspective | HTML
Divergence, Convergence, and Therapeutic Implications: A Cell Biology Perspective of C9ORF72-ALS/FTD | Molecular Neurodegeneration | Full Text
Pathogenesis Resulting from the C9ORF72 Noncoding GGGGCC Expansion in... | Download Scientific Diagram
Biomedicines | Free Full-Text | Disease Mechanisms and Therapeutic Approaches in C9orf72 ALS-FTD | HTML
Pathogenic Mechanisms and Therapy Development for C9orf72 Amyotrophic Lateral Sclerosis/Frontotemporal Dementia | SpringerLink
Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models | Nature Communications
IJMS | Free Full-Text | Gene Therapy for ALS—A Perspective | HTML
Representation of the pathological mechanisms involved in C9orf72-ALS.... | Download Scientific Diagram
Oligonucleotide-Based Therapy for FTD/ALS Caused by the C9orf72 Repeat Expansion: A Perspective
There has been an awakening: Emerging mechanisms of C9orf72 mutations in FTD/ALS - ScienceDirect
Biogen and Ionis Announce Topline Phase 1 Study Results of Investigational Drug in C9orf72 Amyotrophic Lateral Sclerosis
There has been an awakening: Emerging mechanisms of C9orf72 mutations in FTD/ALS - ScienceDirect
Frontiers | The Development of C9orf72-Related Amyotrophic Lateral Sclerosis and Frontotemporal Dementia Disorders | Genetics
Molecular Mechanisms of Neurodegeneration Related to C9orf72 Hexanucleotide Repeat Expansion
New gene therapy targeting C9orf72-ALS begins Phase 1 clinical trial in the UK - MND Research Blog
Gene therapy for ALS: A review - ScienceDirect
Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration | PNAS
Pathogenic Mechanisms and Therapy Development for C9orf72 Amyotrophic Lateral Sclerosis/Frontotemporal Dementia | SpringerLink
Emerging antisense oligonucleotide and viral therapies for amyotrophic lateral sclerosis. - Abstract - Europe PMC
