Innovative System Breaks Barriers in Brain Drug Delivery

Researchers at the Icahn School of Medicine at Mount Sinai have introduced an innovative blood-brain barrier-crossing conjugate (BCC) system, crucial for delivering larger therapeutic molecules into the brain. **This system leverages γ-secretase-mediated transcytosis** to administer oligonucleotides and proteins directly into the central nervous system via intravenous injections, bypassing the protective blood-brain barrier which also restricts drug delivery. **The breakthrough was demonstrated using BCC10, linked to antisense oligonucleotides**, which successfully reduced harmful gene activity in brain tissues. In ALS mouse models, BCC10 decreased the expression of the Sod1 gene, while also reducing the tau protein gene (Mapt) in Alzheimer's research. These findings, published in _Nature Biotechnology_, highlight the potential for efficiently silencing harmful genes in various neurological conditions. As the treatment showed minimal side effects in mice, the investigators plan additional studies in larger animal models to further develop the platform's therapeutic capabilities. Co-authors Yizhou Dong and Eric J. Nestler emphasize that overcoming the blood-brain barrier with this system could revolutionize treatments for numerous brain diseases.