Opening The Blood-Brain Barrier For Gene Therapy

The blood-brain barrier is a lipid-based protective filter that lines the blood vessels serving the brain. It prevents the entry of harmful chemicals and microorganisms. Now, Rice University bioengineers report the use of highly focused ultrasound to non-invasively poke multiple micro-holes in this barrier in order to admit a gene-carrying adenovirus capable of modifying brain cells in a beneficial way. Using a mouse model, the researchers opened up as many as 105 portals and demonstrated successful gene editing in the targeted brain regions. Multiple viral entry points are necessary since each gateway only permits access to a few millimeters of brain tissue. This technique remains in its infancy, but this proof-of-concept opens the door for possible genetic management of degenerative brain disease ……..someday soon.

https://www.nature.com/articles/s41434-023-00421-1

#brain #bloodbrainbarrier #ultrasound #genetherapy #viralvector