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shREDD1-AAV
- Development stage
- Preclinical
- Lead developer
- Penn State College of Medicine
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
- Administration
- Parenteral
01
Overview
shREDD1-AAV is an adeno-associated virus (AAV) vector-based gene therapy designed to deliver short hairpin RNA (shRNA) to suppress the expression of REDD1 (Regulated in Development and DNA Damage Response 1). REDD1 is a stress-induced protein that, when elevated in podocytes under diabetic conditions, drives mitochondrial dysfunction, ferroptosis, and innate immune signaling via the cGAS-STING pathway. By silencing REDD1 specifically in podocytes, this therapeutic candidate aims to prevent podocyte loss, reduce renal inflammation, and halt the progression of diabetic nephropathy (DN). Preclinical studies in diabetic mouse models (db/db) have demonstrated that systemic administration of shREDD1-AAV attenuates albuminuria and preserves renal function.
- Other names
- AAV-shREDD1AAV-shREDD-1AAV-shREDD 1
02
Targets
DDIT4 (Regulated in development and DNA damage response protein 1)DDIT4 (DNA damage-inducible transcript 4 protein)
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