Blood biomarkers to monitor neuroinflammation: Insights from hematopoietic stem cell transplantation and gene therapy in X-linked adrenoleukodystrophy
Isabelle Weinhofer, Markus Ponleitner, Paulus Rommer, Wolfgang Köhler, Jörn-Sven Kühl · FENS Forum 2024
Cerebral adrenoleukodystrophy (CALD) is an X-linked inflammatory demyelinating disorder leading to death within a few years. CALD can be halted by hematopoietic stem cell transplantation (HSCT) or gene therapy (GT) when applied at early disease stages. Currently, eligibility for treatment is determined by MRI-based brain lesion Loes scoring. However, this grading system poorly reflects later disease stages or atypical lesion patterns. As such, there is a need for alternative biomarkers to help with treatment decisions. We recently identified inflammatory activity and CALD progression being ref
ePoster · Neuroscience