rAAV-TH

rAAV-TH represents a gene therapy approach utilizing a recombinant adeno-associated virus (rAAV) vector to deliver the gene encoding tyrosine hydroxylase (TH) into target cells, typically in the brain. The rAAV vector, a modified, non-pathogenic virus, enters cells via receptor-mediated endocytosis and transports its single-stranded DNA genome to the nucleus. Once inside the nucleus, the delivered TH gene is expressed, leading to the production of the tyrosine hydroxylase enzyme. In the context of Parkinson's disease, where dopamine production is impaired, the aim is for the expressed TH enzyme to catalyze the rate-limiting step in dopamine synthesis, thereby increasing local dopamine levels in affected brain regions like the striatum. This approach seeks to restore dopaminergic function and alleviate motor symptoms associated with the disease.

Other names

Recombinant adeno-associated virus-tyrosine hydroxylaseAAV-TH