Researchers at the Icahn School of Medicine at Mount Sinai have developed an innovative approach-;demonstrated in mouse models and isolated human brain tissue-;to safely and effectively deliver therapeutics into the brain, providing new possibilities for treating a wide range of neurological and psychiatric diseases.
Mount Sinai Health SystemNov 25 2024
The BCC platform takes advantage of a specialized biological process called γ-secretase-mediated transcytosis to deliver large therapeutic molecules, like oligonucleotides and proteins, directly into the brain through a simple intravenous injection. The study showed that when the researchers injected a compound called BCC10 linked to specialized genetic tools known as antisense oligonucleotides into mice, it successfully reduced the activity of harmful genes in the brain. In a transgenic mouse model of ALS , the treatment significantly lowered levels of the disease-causing gene called Sod1 and its associated protein.
"Our platform could potentially solve one of the biggest hurdles in brain research-;getting large therapeutic molecules past the blood-brain barrier safely and efficiently," says co-corresponding senior author Eric J. Nestler, MD, PhD, Nash Family Professor of Neuroscience, Director of The Friedman Brain Institute, and Dean for Academic Affairs of Icahn Mount Sinai, and Chief Scientific Officer of the Mount Sinai Health System.
Brain Addiction Alzheimer's Disease Amyotrophic Lateral Sclerosis Antisense Biotechnology Central Nervous System Drugs Gene Genes Genetic Genomics Hospital Immunology Medicine Nervous System Neuroscience Oligonucleotides Protein Research Sclerosis Therapeutics
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