Kommentar fra en amatør.
De ser ut til å ha brukt miRNA og AAV5 men den er tilført lokalt via komplisert hjernekirurgi.
CircVec kan tilpasses forskjellige AAV varianter så kanskje kan man muligens kan sette opp et circVec - AAV5???
CircVec gis med injeksjon hittil. Om det kunne erstattet kirurgi hadde det vært noe. Men dette er nok ikke aktuelt fokus for Circio nå. Det illustrerer genterapiens potensiale fremover og bruk av AAV.
AMT-130 is the very first gene therapy designed specifically for HD that has made it into human clinical trials. Instead of being taken as a pill or an injection, AMT-130 is delivered directly into the brain through a surgical procedure. uniQure believes that AMT-130 has the potential to be a treatment that lasts for life.
"AMT-130 is packaged in a specially-designed harmless virus called AAV5. Think of this virus like a Trojan Horse – a shell used as a package to deliver something (good this time!) into the brain. This virus contains the blueprints to make a special genetic molecule that sticks to the instructions cells normally use to make the huntingtin protein. By binding to these instructions, AMT-130 essentially marks them for destruction. With fewer instructions around, cells make less huntingtin protein overall, including the harmful version linked to HD. The treatment lowers levels of both the expanded and regular huntingtin protein.
Brave steps towards a gene therapy for HD
The effects of gene therapies, like AMT-130, are irreversible and its delivery by brain surgery carries many risks."