Gene-based drug administration using the process by which cells transport substances on their own
input 2024.11.28 20:48
input 2024.11.28 20:48
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Animal testing to deliver gene therapy to the brain has been successful. [사진=게티이미지뱅크]There is a hard cell layer called the blood-brain barrier on the inner surface of the cerebral blood vessels surrounding brain cells. The blood-brain barrier prevents bacteria, viruses, and toxins from entering the brain by filtering out large molecules that can enter the brain through the bloodstream, but it also makes it difficult for drugs to get to the brain.
An animal experiment was successful in breaking through this barrier using natural transport functions and delivering gene therapy to the mouse brain. This is what the health and medicine webzine ‘Health Day’ reported on the 27th (current time) based on a paper by researchers at the Icahn School of Medicine at Mount Sinai Hospital in the U.S. published in Nature Biotechnology.
The treatment successfully reduced the activity of harmful genes in the brains of laboratory mice that cause Lou Gehrig’s disease, Alzheimer’s disease and dementia, the researchers said. “The blood-brain barrier is an essential defense mechanism, but it poses a significant challenge in delivering drugs to the brain,” said lead researcher Zhou Dong Lee, an immunology professor at the Icahn School of Medicine. “This new technology, called the blood-brain barrier cross-conjugate (BCC) system, is This “breaks down this barrier,” he explains, “allowing the drug to safely and efficiently reach the central nervous system.”
The BCC system uses the process of transcytosis, in which cells transport substances themselves. Substances are captured in cells, move through the cell interior, and are then released on the other side.
The researchers said in the paper that they were able to link the gene-based drug to a compound called BCC10 and inject it into the bloodstream of mice. The BCC10 compound uses transcytosis to induce cells in the blood-brain barrier to internally transport drugs across the barrier. The treatment was well tolerated in mice, causing little or no damage to major organs, the researchers noted.
“Our platform can potentially solve one of the biggest obstacles in brain research: getting large therapeutic molecules to safely and efficiently cross the blood-brain barrier,” said co-principal investigator Eric Nessler, director of the Friedman Brain Institute at Mount Sinai Hospital. “This development has the potential to advance treatments for a wide range of brain diseases.” In the future, the researchers plan to examine the BCC system in larger animals to verify its function and develop its potential to treat diseases.
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