clinical 977 words KG: ent-gene-9d811bdb 2026-03-28
kind:clinical-trialsection:clinical-trialsstate:preclinicaltopic:stxbp1-encephalopathytopic:gene-therapytopic:aaav
Contents

STXBP1 Encephalopathy — Preclinical Gene Therapy Program

🔬 Protein Info
Gene Symbolent-gene-9d811bdb
Target IndicationSTXBP1 Encephalopathy (EIEE5)
GeneSTXBP1
Gene Size~2 kb coding sequence
VectorAAV (serotype TBD)
Delivery RouteIntrathecal or intracisterna magna (under investigation)
Development StagePreclinical (research/lead optimization)
Lead GroupsMultiple academic laboratories
KG Connections2 knowledge graph edges
DatabasesGeneCardsUniProtNCBI GeneHPASTRING

Knowledge Graph

Related Hypotheses (28)

Bacterial Enzyme-Mediated Dopamine Precursor Synthesis
Score: 0.36
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
PARP1 Inhibition Therapy
Score: 0.58

Related Analyses (16)

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Autophagy-lysosome pathway convergence across neurodegenerat
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What are the mechanisms by which gut microbiome dysbiosis in
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Related Experiments (1)

ER-Golgi Secretory Pathway Dysfunction in PD - Experiment De
clinical · proposed · Score: 0.40

Knowledge Graph (2 edges)

ent-gene-9d811bdb data_in benchmark_ot_ad_answer_key:STXBP1
benchmark_ot_ad_answer_key:STXBP1 data_in ent-gene-9d811bdb