• Gene therapy for Alzheimer’s disease is an experimental treatment that involves the modification of brain cells with specific genes that are programmed to produce therapeutic molecules.
  • The blood-brain barrier (BBB) prevents adequate gene delivery to brain cells.
  • Researchers at Sunnybrook Research Institute tested whether focused ultrasound can be used to improve gene delivery across the BBB.

Key Points

  • Gene therapy for Alzheimer’s disease is an experimental treatment that involves the modification of brain cells with specific genes that are programmed to produce therapeutic molecules.
  • The blood-brain barrier (BBB) prevents adequate gene delivery to brain cells.
  • Researchers at Sunnybrook Research Institute tested whether focused ultrasound can be used to improve gene delivery across the BBB.

Gene Therapy for AD imageGene therapy for Alzheimer’s disease is an experimental treatment that involves the modification of brain cells with specific genes that are programmed to produce therapeutic molecules. Gene therapy can be used to repair, replace, add, or remove genes to fight disease. Gene carriers are used to deliver the genes to the target cells. Several clinical trials have been launched to test whether gene therapy can provide a treatment option for patients with Alzheimer’s disease and other neurodegenerative disorders. In the brain, however, the blood-brain barrier (BBB) prevents adequate gene delivery by blocking the gene carriers injected into the blood and preventing them from reaching the brain cells. A discovery that has improved gene delivery to the brain is the engineering of the gene carriers that can, to some extent, cross the BBB. These engineered gene carriers are promising, but there is more work to be done.

Researchers at Sunnybrook Research Institute are testing whether focused ultrasound can be used to improve gene delivery across the BBB. Rikke Hahn Kofoed, PhD, and her team in the Aubert Laboratory, recently published a preclinical study that describes this new, noninvasive technique, which improved the delivery of gene carriers across the BBB and into the brain in a mouse model of Alzheimer’s pathology. Specifically, focused ultrasound combined with intravenous microbubbles increased the permeability of the BBB at targeted sites and allowed the new gene carriers to cross the BBB. With an eye toward the safety of the technique, the authors also described how their noninvasive delivery of genes to the brain elicited an immune response that was mild and controllable, which is similar to what happens when gene carriers are directly injected into the brain. See Molecular Therapy Methods & Clinical Development >

Sunnybrook recently interviewed Drs. Kofoed and Aubert to learn more. See “Behind the research: Study explores delivery of gene therapy to treat Alzheimer’s” >

Photo: Gene delivery image courtesy of Rikke Hahn Kofoed, PhD, and the Aubert Laboratory at Sunnybrook Research Institute.

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