Experimental gene therapy could one day treat hydrocephalus The first proof-of-concept study showed that the gene therapy reduced the production of cerebrospinal fluid in the brains of mice. Written byAllison Whitten, PhD

Experimental gene therapy could one day treat hydrocephalus The first proof-of-concept study showed that the gene therapy reduced the production of cerebrospinal fluid in the brains of mice. Written byAllison Whitten, PhD https://www.drugdiscoverynews.com/experimental-gene-therapy-could-one-day-treat-hydrocephalus-17589?utm_campaign=DDN_Newsletter_Dose&utm_medium=email&_hsenc=p2ANqtz-8ohNvHT9U8QmyV8VQdS_g85lHmXQ8RgxODkA8m4SEQo3ckMnrzOYOLhnbTvkngP3LV8Pyd6BJUEOZVpin3T5uh5qfheg&_hsmi=444434988&utm_content=444434988&utm_source=hs_email When too much cerebrospinal fluid (CSF) seeps into the cavities of the brain, the result is life-threatening hydrocephalus. As the fluid builds up, it puts pressure around the surrounding fragile brain tissue. The primary treatment option involves an invasive surgery that inserts a shunt to drain the fluid elsewhere in the body, though these can often fail down the line. New research from Yu Luo’s lab at the University of Cincinnati showed a new way forward that gets directly at the source. By delivering a new AAV gene therapy into CSF that targets the choroid plexus — the network of vascular tissue that produces CSF — the researchers reduced CSF in a mouse model of hydrocephalus.