therapeutic 1,174 words KG: ent-gene-9f063e98
kind:therapeuticsection:therapeuticsstate:publishedtopic:parkinsons
Contents

LRRK2 Antisense Oligonucleotide Therapies for Parkinson's Disease

🔬 Protein Info
Gene Symbolent-gene-9f063e98
Target RegionRationale
Coding sequenceDisrupt translation initiation or elongation
Splice sitesAlter splicing to create non-functional transcripts
3' UTRAffect mRNA stability and localization
TargetLRRK2 mRNA
CompanyBristol Myers Squibb
Chemistry2'-O-methoxyethyl (2'-MOE)
RouteIntrathecal (spinal) injection
PhaseDiscovery / Preclinical
NCT NumberNCT03976375
IndicationParkinson's disease
AspectLRRK2 Kinase Inhibitors
MechanismEnzymatic inhibition
Protein levelsUnchanged
DosingOral (small molecule)
KG Connections2 knowledge graph edges
DatabasesGeneCardsUniProtNCBI GeneHPASTRING

Knowledge Graph

Related Hypotheses (30)

Bacterial Enzyme-Mediated Dopamine Precursor Synthesis
Score: 0.36
Circadian-Synchronized LRP1 Pathway Activation
Score: 0.53
Gamma entrainment therapy to restore hippocampal-cortical sy
Score: 0.68
Selective Acid Sphingomyelinase Modulation Therapy
Score: 0.65
CYP46A1 Overexpression Gene Therapy
Score: 0.63

Related Analyses (30)

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neurodegeneration · completed
Sleep disruption as cause and consequence of neurodegenerati
neurodegeneration · completed
Synaptic pruning by microglia in early AD
neurodegeneration · completed
Epigenetic clocks and biological aging in neurodegeneration
neurodegeneration · completed
TDP-43 phase separation therapeutics for ALS-FTD
neurodegeneration · archived

Related Experiments (30)

AAV Serotype Comparison for LRRK2 Knockdown in PD Gene Thera
validation · proposed · Score: 0.40
AAV-LRRK2 IND-Enabling Study Design
clinical · proposed · Score: 0.40
AAV Serotype Comparison for LRRK2 Knockdown in PD
validation · proposed · Score: 0.40
Alpha-Synuclein Aggregation Triggers — Sporadic PD Initiatio
clinical · proposed · Score: 0.40
Computational Modeling of Alpha-Synuclein Propagation in PD
validation · proposed · Score: 0.40

Knowledge Graph (2 edges)

ent-gene-9f063e98 data_in benchmark_ot_ad_answer_key:LRRK2
benchmark_ot_ad_answer_key:LRRK2 data_in ent-gene-9f063e98