Key Points

  • Scientists at the University of Virginia explored the effect of varying focused ultrasound energy levels to deliver gene therapy to the brain.
  • The researchers were able to fine tune the focused ultrasound to preferentially deliver genetic material to specific types of brain cells.
  • Various pressure levels enabled the genes to reach cells within and beyond the blood-brain barrier.

When using focused ultrasound to disrupt the blood-brain barrier (BBB) and deliver gene therapy, scientists at the University of Virginia explored the effects of increasing or decreasing the energy level, or peak negative pressure (PNP), on determining which cells received the genetic material transfer. In “Single-Cell Mapping of Focused Ultrasound―Transfected Brain,” they found that the focused ultrasound could be tuned to preferentially deliver genetic material to many different types of cells within the brain. For example, at 0.2 MPa PNP, the endothelial cells, pericytes, and astrocytes in the BBB were enriched, and at 0.4 MPa PNP, the neurons, oligodendrocytes, and microglia that are distal to the BBB were augmented. What happened to the cell stress genes?

See Gene Therapy >

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