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AAV-tyrosinase
- Development stage
- Preclinical
- Lead developer
- Universidad de Navarra
- Modality
- Gene Therapies
- Administration
- Intravitreal, Parenteral
01
Overview
AAV-tyrosinase is an adeno-associated virus (AAV)-based gene therapy approach investigated in preclinical research for the treatment of oculocutaneous albinism type 1 (OCA1) and the development of Parkinson's disease models. In the context of OCA1, the vector delivers a functional human tyrosinase (TYR) gene to ocular tissues, particularly the retinal pigment epithelium, to restore melanin synthesis and prevent photoreceptor degeneration. In Parkinson's disease research, AAV-tyrosinase is used to drive the overexpression of tyrosinase in the substantia nigra, leading to the accumulation of neuromelanin and subsequent synucleinopathy, which mimics the neuropathology of the disease in non-human primates.
- Other names
- AAV-mediated tyrosinase
02
Targets
TYR (Tyrosinase)HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))
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