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AAV-SIRT3
- Development stage
- Preclinical
- Lead developer
- Kyungpook National University
- Modality
- Gene Therapies
- Administration
- Intravenous, Parenteral
01
Overview
AAV-SIRT3 is an adeno-associated virus (AAV) vector-based gene therapy designed to overexpress Sirtuin 3 (SIRT3), a mitochondrial NAD+-dependent deacetylase. SIRT3 plays a critical role in maintaining mitochondrial function, regulating oxidative stress, and promoting cellular survival. Preclinical studies have investigated AAV-SIRT3 for various indications, including Parkinson's disease, sarcopenia (age-related muscle wasting), metabolic dysfunction-associated steatohepatitis (MASH), and cognitive impairment. By delivering the Sirt3 gene directly to target tissues, such as the substantia nigra or hippocampus, AAV-SIRT3 aims to restore mitochondrial bioenergetics, reduce oxidative stress, and prevent neurodegeneration or tissue damage.
- Other names
- AAV-Sirt3AAV-Sirt-3AAV-Sirt 3AAV-SIRT3-mycAAV-SIRT-3-mycAAV-SIRT 3-mycAAV.SIRT3-mycAAV1-SIRT3AAV-1-SIRT3AAV 1-SIRT3rAAV1.SIRT3-mycrAAV-1.SIRT3-mycrAAV 1.SIRT3-myc
02
Targets
BNIP3 (BCL2/adenovirus E1B 19 kDa protein-interacting protein 3)CPT2 (Carnitine O-palmitoyltransferase 2)FOXO3 (Forkhead box protein O3)GSK3 (Glycogen synthase kinase 3 beta)NDUFA9 (NADH dehydrogenase [ubiquinone] 1 alpha subcomplex subunit 9)PPIF (Peptidyl-prolyl cis-trans isomerase F)SIRT3 (NAD-dependent protein deacetylase sirtuin-3)Misfolded SOD1
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