080210xf's Blog

L'X fragile sera vaincu | Fragile X will be conquered

Cell’s own machinery can deliver therapies to the brain

Jessica Wright, Sfari.org |

Exosomes, the brain’s system for delivering and recycling molecules, can be manipulated to carry therapeutic fragments of RNA or DNA across the blood-brain barrier and into neurons. The ingenious new technique was published 20 March in Nature Biotechnology.

As studies uncover the genetic and molecular causes of autism, gene therapy — silencing the expression or boosting levels of a particular gene product — could emerge as a therapeutic option.

Targeting these genes to brain cells is complicated, however. Injecting constructs into the blood delivers them indiscriminately to all tissues, and the blood-brain barrier prevents most molecules from crossing into the brain at all.

In the new study, researchers solved both problems by coating mouse exosomes — bubbles of membrane that pinch off and merge their contents when they fuse with another membrane — with a brain-targeting protein.


No comments yet»

Leave a Reply

Fill in your details below or click an icon to log in:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out /  Change )

Google photo

You are commenting using your Google account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )

Connecting to %s

%d bloggers like this: