Scientists at the Icahn School of Medicine at Mount Sinai have developed a lipid nanoparticle system capable of delivering messenger RNA (mRNA) to the brain via intravenous injection, a challenge that has long been limited by the protective nature of the blood-brain barrier.
The findings, in mouse models and isolated human brain tissue, were published in the February 17 online issue of Nature Materials [10.1038/s41563-024-02114-5]. They demonstrate the potential of this technology to pave the way for future treatments for a wide range of conditions such as Alzheimer’s disease, amyotrophic lateral sclerosis, brain cancer, and drug addiction.Â
The blood-brain barrier serves as a protective shield, preventing many substances-including potentially beneficial therapies-from reaching the brain. While previous research from Mount Sinai introduced a platform for transporting large biomolecules such as proteins and oligonucleotides into the central nervous system, this new study focuses on a different approach: using specially designed lipid nanoparticles to transport mRNA across the barrier.
Getting mRNA into the brain could allow scientists to instruct brain cells to produce therapeutic proteins that can help treat or prevent disease by replacing missing proteins, reducing harmful ones, or activating the body’s defenses.
Our study shows that these blood-brain barrier-crossing lipid nanoparticles (BLNPs) can safely and efficiently deliver mRNA into the brain. This could open up opportunities to use mRNA-based therapies for a variety of neurological and psychiatric disorders.”
Yizhou Dong, PhD, co-corresponding senior author, Professor of Immunology and Immunotherapy, and member of the Icahn Genomics Institute and the Marc and Jennifer