THERAPY DOSSIER · th-224f8243ff32

Lentiviral vector-delivered aquaporin-4 (AQP4) short hairpin RNA

Biologic · Lentiviral aquaporin-4 short-hairpin RNA; RNAi gene therapy.

Evidence & regulatory context

Mechanism: Lentiviral aquaporin-4 short-hairpin RNA; RNAi gene therapy.
Target: Not recorded
Injury models: TBI
Reported indication: Not recorded
First approval: Not recorded
Development stage: Not recorded
ChEMBL record: Not recorded
ChEMBL max phase: Not recorded
Synonyms: Not recorded
Note: Not recorded

Clinical trials 0

0 linked trials carry an injury-indication flag. These flags support record-level exploration and sit alongside the stringent aggregate counts described in Methods.

No linked human trial in this dataset.

Preclinical evidence 1

1 studies reported at therapy level; 1 evidence entries itemised.

PMID 30246455 ↗2018Reported benefit

Inhibitory role of lentivirus-mediated aquaporin-4 gene silencing in the formation of glial scar in a rat model of traumatic brain injury.

RatTBIUnspecifiedSprague-DawleyMaleAfter establishment of the traumatic brain injury model

Model: Rat model of traumatic brain injury, specific induction method not stated in abstract

At 7, 14 and 28 days after injury, rats treated with AQP4 shRNA showed improved neurological function and reduced histomorphological change, with decreased mRNA and protein expression of GFAP, vimentin, fibronectin and laminin and reduced positive cell counts, positive area ratio and glial scar for…

Extraction: extract · Supplied extraction confidence: 0.5. Machine-extracted fields require source verification.

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