“Special Delivery” could be the theme for this month’s research highlights. Whether it is gene therapy or antibodies, brain disorders or bone growth, magnetic resonance imaging or ultrasound guidance, biomedical researchers are finding focused ultrasound to be the tool that enables therapeutic molecules to reach diseased cells like never before. The studies cover Parkinson’s disease, Alzheimer’s disease, and nonunion bone fractures.
Novel Focused Ultrasound Gene Therapy Approach Noninvasively Restores Dopaminergic Neuron Function in a Rat Parkinson’s Disease Model
The Price/Hanes collaboration for nanoparticle fabrication and delivery made a breakthrough in a rat model of Parkinson’s disease. Their treatment strategy of using focused ultrasound to deliver brain-penetrating nanoparticles across the blood-brain barrier resulted in widespread but targeted glial cell—derived neurotrophic factor (GDNF) transgene expression. See Nano Letters.
Amyloid β Plaque Reduction with Antibodies Crossing the Blood-Brain Barrier, Which was Opened in 3 Sessions of Focused Ultrasound in a Rabbit Model
Delivering antibodies with ultrasound guidance, researchers at the Cyprus University of Technology have shown that using focused ultrasound to open the blood-brain barrier reduces amyloid B plaque in the rabbit brain. See the Journal of Ultrasound in Medicine.
In Situ Bone Tissue Engineering Via Ultrasound-mediated Gene Delivery to Endogenous Progenitor Cells in Mini-pigs
In a truly collaborative study involving four centers in the United States and Israel, scientists used focused ultrasound and microbubbles to deliver therapeutic genes to endogenous stem cells in a large animal model. This treatment led to complete fracture healing 6 weeks after treatment versus nonunion in control animals. See Science Translational Medicine for the study and see animation of the experiment in Science Magazine.