Research ArticleGene Therapy

Gene therapy delivering a paraoxonase 1 variant offers long-term prophylactic protection against nerve agents in mice

See allHide authors and affiliations

Science Translational Medicine  22 Jan 2020:
Vol. 12, Issue 527, eaay0356
DOI: 10.1126/scitranslmed.aay0356

Long-lasting protection

Nerve agents are the most toxic and lethal warfare chemical compounds. The toxic effect is due to inhibition of the acetylcholinesterase enzymes. Current antidotes prevent death but are ineffective against complications including brain damage and behavioral impairments. To prevent these deleterious consequences, now, Betapudi et al. developed a prophylactic gene therapy delivering the gene coding for a variant of the nerve agent scavenger paraoxonase 1 (PON1). When injected systemically in mice, the treatment exerted long-lasting protection against different types of nerve agents without signs of toxicity. The results suggest that gene therapy might be a viable option as prophylactic treatment in subjects at risk of exposure to nerve agents such as military personnel in war zones.

View Full Text

Stay Connected to Science Translational Medicine