In a recent study published in Advanced Materials, a team of researchers at the University of Wisconsin-Madison have developed a novel method to transport nan-scale capsules to deliver brain-wide therapies that can be used to treat neurological disorders, such as Alzheimer’s and Parkinson’s.
"There is no cure yet for many devastating brain disorders," Dr. Shaoqin "Sarah" Gong, a UW-Madison professor of ophthalmology, who is also a visual sciences and biomedical engineering and researcher at the Wisconsin Institute for Discovery, and a co-author on the study, said in a statement. "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."
For the study, led by postdoctoral researcher, Dr. Yuyuan Wang, the research team describe the use of nano-scale capsules comprised of silica capable of delivering gene-editing tools to the brains of mice and then harmlessly dissolve once the medication of treatment is delivered. The researchers state that a higher therapeutic efficacy is achievable since the nanoparticles can be delivered both intravenously and repeatedly.
The team hopes to improve on this study by in order to treat a variety of brain disorders, while also being examined for delivering biologics to the eyes, lungs, and liver, which could possibly lead to novel gene therapies for various other disorders, as well.
"The promise of brain gene therapy and genome-editing therapy relies on the safe and efficient delivery of nucleic acids and genome editors to the whole brain," said Dr. Gong.
Sources: Advanced Materials, UW-Madison News, The Jackson Laboratory
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