A NOTCH3 Pathogenic Variant Influences Osteogenesis and Can Be Targeted by Antisense Oligonucleotides in Induced Pluripotent Stem Cells

To determine the consequences of Lateral Meningocele Syndrome pathogenic variants in human cells and whether they can be targeted by antisense oligonucleotides, induced pluripotent NCRM1 and NCRM5 stem cells harboring a NOTCH36692-93insC insertion were created.