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AAV-leptin
- Development stage
- Preclinical
- Lead developer
- Massachusetts Institute of Technology
- Modality
- Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
- Administration
- Intramuscular, Intraperitoneal
01
Overview
AAV-leptin is an experimental gene therapy construct designed to deliver a functional leptin (LEP) gene using a recombinant adeno-associated virus (AAV) vector. It is primarily used in preclinical research to restore circulating leptin levels in models of congenital leptin deficiency, such as ob/ob mice. By providing a sustained source of leptin, the therapy aims to normalize metabolic abnormalities, including obesity, hyperglycemia, and insulin resistance. Research has also explored its role in intercepting obesity-mediated cancer progression, particularly in pancreatic cancer models. Various serotypes and targeting variants, such as adipose-targeting Rec2-leptin, have been investigated across multiple academic institutions.
- Other names
- recombinant adeno-associated virus-leptinAAV-LEP
02
Targets
LEPR (Leptin receptor)
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