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AAV9-RBPMS
- Development stage
- Preclinical
- Lead developer
- Xinhua Hospital
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV9-RBPMS is an experimental gene therapy candidate utilizing an adeno-associated virus serotype 9 (AAV9) vector to deliver the gene encoding RNA-binding protein with multiple splicing (RBPMS). Developed for the treatment of heart failure with preserved ejection fraction (HFpEF), the therapy aims to counteract the downregulation of RBPMS caused by neutrophil elastase (NE). RBPMS is a critical regulator of Titin mRNA alternative splicing; its restoration via AAV9-mediated cardiac overexpression is intended to normalize Titin isoforms, thereby improving myocardial compliance and alleviating diastolic dysfunction.
- Other names
- adeno-associated virus-9-mediated RBPMSAAV9-mediated RBPMS cardiac overexpressionAAV-9-mediated RBPMS cardiac overexpressionAAV 9-mediated RBPMS cardiac overexpression
02
Targets
RBPMS (RNA binding protein with multiple splicing)
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