Autologous Hematopoietic Stem Cell Gene Therapy With Ex-Vivo LV Platform in MPS II

Autologous Hematopoietic Stem Cell Gene Therapy With Ex-Vivo LV Platform in MPS II by Madaline Spencer and James Radke, PhD| Published on: Mar 12, 2026 Brian Bigger, PhD, Professor of Advanced Therapeutics at the University of Edinburgh, discusses the first-in-human experience of autologous hematopoietic stem cell gene therapy (HSCGT) using a novel ex-vivo lentiviral (LV) platform to correct mucopolysaccharidosis II (MPS II). https://checkrare.com/autologous-hematopoietic-stem-cell-gene-therapy-with-ex-vivo-lv-platform-in-mps-ii/