Synthetic nanoparticles for gene editing in the brain in utero

Synthetic nanoparticles for gene editing in the brain in utero
2014 Scientific Innovations Award
W. Mark Saltzman, Ph.D.
Department of Biomedical Engineering
Yale University

Dr. Saltzman and his lab propose an innovative and potentially transformative approach for the correction of single-gene disorders of the central nervous system in the fetal brain: in utero administration of synthetic nanoparticles carrying triplex-forming oligonucleotides, which they have shown to be a powerful gene-editing agent. They will optimize nanoparticle size, chemistry, and composition for targeted delivery to fetal brain cells after intra-uterine administration. Dr. Saltzman and his lab will test the effectiveness of this approach by attempting to correct the gene disorder in Hurler Syndrome in mice. If they are successful, this novel approach can be applied to any monogenic gene defect in the brain. Further, our nanoparticles—which will be produced from biocompatible materials -can potentially by used for the in utero delivery of any agent and therefore may be useful for treating a wide ranges of diseases in the brain.