Gene editing through the blood-brain barrier using focused ultrasound

The blood-brain barrier (BBB) remains a significant challenge to the therapy of brain disorders and diseases.

The blood-brain barrier’s job is to protect the brain from toxins and pathogens that are detrimental to the brain. However, this makes it difficult to introduce drugs to treat neurological conditions and cancers of the brain.

When scientists discovered in the early 2000s that they can use ultrasound to open the blood-brain barrier, this led to the idea that ultrasound can be used in conjunction with therapeutics to allow them to reach the brain.

This note describes the decades-long history of research into the blood-brain barrier, the advancements in ultrasound technologies, and how they can be used to breach the blood-brain barrier to deliver therapeutics.

Dive into the note for more details:

• What makes the blood-brain barrier difficult to cross

• How focused ultrasound technologies work

• How focused ultrasound enables gene editing in the brain via intravenous AAV9