This technology is a CRISPR-based approach for specific removal of a chromosome for prevention of trisomies (chromosome gains).
Errors in the production of germ cells can cause mis-segregation of genetic material, known as aneuploidy. Germ cell aneuploidy dramatically increases with advanced maternal age, leading to miscarriages, genetic defects, and reduced fertility. Current technology enables aneuploid embryos to be identified and eliminated in in vitro fertilization clinics. However, there is currently no corrective therapy for these embryos, making aneuploidy an insurmountable reproductive obstacle for some patients. Recently, CRISPR/Cas9 gene editing has emerged as a promising method to modify chromosome content at the time of conception without discarding embryos. This molecular tool has yet to be harnessed for the specific removal of a chromosome for disease-correcting applications.
This technology is a CRISPR-based strategy for removing a disease-causing supernumerary chromosome at and shortly after fertilization. This method requires one cut by Cas9, which can be directed in an allele-specific manner, to eliminate an entire chromosome arm with a high efficiency of approximately 50% with a single guide. The combination of several guides is expected to increase efficiency of removal.
Patent Pending(US17/717,697)
IR CU20109, CU20250
Licensing Contact: Kristin Neuman
