Adeno-associated virus-mediated expression of human butyrylcholinesterase to treat organophosphate poisoning.

ABSTRACT: Rare diseases defined by genetic mutations are classic targets for gene therapy. More recently, researchers expanded the use of gene therapy in non-clinical studies to infectious diseases through the delivery of vectorized antibodies to well-defined antigens. Here, we further extend the utility of gene therapy beyond the "accepted" indications to include organophosphate poisoning. There are no approved preventives for the multi-organ damage resulting from acute or chronic exposure to organophosphates. We show that a single intramuscular injection of adeno-associated virus vector produces peak expression (~0.5 mg/ml) of active human butyrylcholinesterase (hBChE) in mice serum within 3-4 weeks post-treatment. This expression is sustained for up to 140 days post-injection with no silencing. S

SUBMITTER: Gupta V 

PROVIDER: S-EPMC6876934 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature