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Experiment #00000059
Amyotrophic lateral sclerosis; Biomarkers; Clinical trials; Neuron-derived extracellular vesicles; Neuropathology; Pharmacodynamic biomarkers; Surrogate endpoints; TDP-43
Disease Progression
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Biological Markers
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Proteomics
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Platform Data
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Patient Selection
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TDP-43 pathology
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ALS
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Axonal Injury
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+9 more
View Results
Experiment #00000030
Can AI and single-cell RNA sequencing help map cryptic TDP-43 splicing errors in sporadic ALS to design BBB-penetrant CRISPR gene therapies before neurotoxicity begins?
Single-Cell Analysis
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RNA Splicing
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_gates_from_rna_splicing
Blood-Brain Barrier
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_gates_from_blood_brain_barrier
Neurotoxicity Syndromes
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TDP-43 pathology
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Exons
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_gates_from_exons
_gates_to_single_cell_analysis
Artificial Intelligence
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DNA-Binding Proteins
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Sequence Analysis, RNA
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CRISPR-Cas Systems
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+15 more
View Results
Experiment #00000023
Does misfolded TDP43 affect Retinal Ganglion Cell STMN2 ability to repair in a similar way as what how it was shown to affect motor neurons with cryptic mis-splicing?
TDP-43 pathology
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RNA Splicing
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Stathmin 2
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Vitreous Body
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DNA-Binding Protein-43
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Retinal Ganglion Cells
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Cell Nucleus
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_gates_from_stathmin_2
Axonal Degeneration
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Axons
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+11 more
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Experiment #00000005
What are the biomarker differences in TDP-43 proteinopathy within the cerebellum and retina when comparing Sporadic Amyotrophic Lateral Sclerosis and c9orf72 affected Familial Amyotrophic Lateral Sclerosis? Are there any mutually exclusive biomarkers that can be deduced?
C9orf72 expansion
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Dipeptide Repeats
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Microglia
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C9orf72 Protein
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Cerebellum
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Sporadic ALS
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TDP-43 Proteinopathies
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Inclusion Bodies
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Amyotrophic Lateral Sclerosis
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Peptides
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C9orf72 Expansion
Atrophy
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TDP-43 Proteinopathy
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Retinal Degeneration
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C9orf72 hexanucleotide repeat
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Transcriptome
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TDP-43 pathology
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Retina
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+26 more
View Results
Reference Abstract
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Source: PubMed
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