iNFixion Bioscience published peer-reviewed paper on haploinsufficiency correction therapy for NF1

iNFixion published a peer-reviewed paper in the Journal of Translational Genetics and Genomics highlighting the rationale for haploinsufficiency correction therapy (HCT) as a potential treatment for neurofibromatosis type 1 (NF1). Evidence supports that a reduced level of functional neurofibromin (NF1 protein) in cells harboring an NF1 mutation plays a direct role in driving NF1 manifestations. By addressing this protein haploinsufficiency, iNFixion’s NF1-HCT offers a promising therapeutic approach by targeting the root cause of NF1.  By enhancing the expression of the NF1 protein from the one normal NF1 gene present in everyone with NF1, iNFixion’s NF1-HCT is predicted to be effective regardless of a person’s specific NF1 mutation. This is critical given that more than 4,000 unique NF1 mutations have already been identified.

Read the full scientific article here.