Therapeutic potential of human stem cell transplantations for Vanishing White Matter: A quest for the Goldilocks graft.

ABSTRACT:

Introduction

Vanishing white matter (VWM) is a leukodystrophy that leads to neurological dysfunction and early death. Astrocytes are indicated as therapeutic target, because of their central role in VWM pathology. Previous cell replacement therapy using primary mouse glial precursors phenotypically improved VWM mice.

Aims

The aim of this study was to determine the translational potential of human stem cell-derived glial cell replacement therapy for VWM. We generated various glial cell types from human pluripotent stem cells in order to identify a human cell population that successfully ameliorates disease hallmarks of a VWM mouse model. The effects of cell grafts on motor skills and VWM brain pathology were assessed.

Results

Transplantation of human glial precursor po

SUBMITTER: Hillen AEJ 

PROVIDER: S-EPMC9344080 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature