Induction of Naturally Occurring Homology Direct Repair in Heterozygous Mutations Using Allele Specific CRISPR Targeting and use of HDR EnhancersHeterozygous mutations cause several genetic disorders. To correct the mutant allele, an HDR (homology directed repair) template is required since HDR initiation does not occur naturally. The IP, a new method to correct heterozygous mutations, does not require an HDR template. By using CRISPR and our specific gRNA design, we can singularly target the mutant allele to initiate HDR repair in heterozygous mutant genes. The IP has demonstrated efficacy in vitro having corrected several mutations, and it has the potential to correct any heterozygous mutation, including CD34+/ bone marrow related disorders and mutant VCP (valosin-containing protein) genes. In the latest stage of development, continued testing is the priority, and parties interested in gene-editing therapies stand to benefit from the IP’s progression. |
![]() Tech ID2023-048 College(None) Licensing ManagerInventorsCategories(None) |