Changing the brain: nanocapsule therapy edits the Alzheimer’s gene
In a recent study, researchers developed a nanocapsule gene therapy that can edit an Alzheimer’s-associated gene in mice, potentially offering a novel treatment for neurological disorders.
One of the greatest limitations in the development of treatments for neurological disorders is the blood–brain barrier (BBB), which stops harmful microorganisms from entering the brain. Although the BBB is normally an important shield against any bacteria, viruses or fungi that may be circulating in blood, it can pose an issue when trying to deliver treatments to the brain.
Researchers at the University of Wisconsin–Madison (WI, USA) have developed a nanocapsule therapy that can bypass the protective BBB membrane and provide a brain-wide, gene-editing therapy that could be beneficial for Alzheimer’s patients one day.
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The research team designed nano-sized capsules made of silica that can carry gene-editing technology, such as CRISPR, into organs and then dissolve following delivery of the therapeutics. They tested these nanocapsules in mice, demonstrating that they were able to penetrate the BBB when their surfaces were modified with glucose and an amino acid derived from the rabies virus. Once the nanocapsules bypassed the BBB, they dissolved and their CRISPR cargo delivered brain-wide gene editing, successfully editing a gene associated with Alzheimer’s disease – amyloid precursor protein.
“There is no cure yet for many devastating brain disorders,” senior author Shaoqin Gong commented. “Innovative brain-targeted delivery strategies may change that by enabling noninvasive, safe and efficient delivery of CRISPR genome editors that could, in turn, lead to genome-editing therapies for these diseases.”
This nanocapsule gene therapy, which is non-invasive and repeatable, allows for the intravenous administration of treatments to the brain. The alternative to bypassing the BBB is injecting treatment directly into the brain; however, this is an invasive procedure that only offers treatment to a small portion of surrounding tissue.
The next step is to optimize the brain-targeting capabilities of the silica nanocapsules and evaluate their potential for treating different brain disorders. Additionally, looking into how this type of treatment delivery technology can be modified to target numerous organs, offering potential treatments for a wide range of diseases.
