Jose-Carlos Segovia, PhD, CIEMAT, CIBERER & IIS- FJD, Madrid, Spain, discusses the limitations associated with the use of adeno-associated virus vectors when targeting primary hyperoxaluria type 1 (PH1), a rare genetic disorder of the liver caused by mutations in the AGXT gene. Given the high integrating capacity of lentiviral vectors, they are being investigated in a preclinical mouse model as a novel therapeutic approach for PH1. Preliminary studies have demonstrated that treatment with hepatocyte-specific lentiviral gene therapy resulted in high levels of gene correction, although further work is required to improve transduction efficiency and enhance therapeutic benefit. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.
Jose-Carlos Segovia is a consultant for and has received research funding from Rocket Pharmaceuticals, Cranbury, NJ.