Taking Advantages of Blood–Brain or Spinal Cord Barrier Alterations or Restoring Them to Optimize Therapy in ALS?

H Alarcan, Y Al Ojaimi, D Lanznaster… - Journal of Personalized …, 2022 - mdpi.com
Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disorder that still
lacks an efficient therapy. The barriers between the central nervous system (CNS) and the …

Hematopoietic stem cell transplantation for neurological disorders: A focus on inborn errors of metabolism

P de Vasconcelos, JF Lacerda - Frontiers in Cellular Neuroscience, 2022 - frontiersin.org
Hematopoietic stem cells have been investigated and applied for the treatment of certain
neurological disorders for a long time. Currently, their therapeutic potential is harnessed in …

Neurofilament light chain as a potential biomarker for monitoring neurodegeneration in X-linked adrenoleukodystrophy

I Weinhofer, P Rommer, B Zierfuss, P Altmann… - Nature …, 2021 - nature.com
Abstract X-linked adrenoleukodystrophy (X-ALD), the most frequent monogenetic disorder of
brain white matter, is highly variable, ranging from slowly progressive …

Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy

A Lauer, SL Speroni, M Choi, X Da, C Duncan… - Nature …, 2023 - nature.com
Blood-brain barrier disruption marks the onset of cerebral adrenoleukodystrophy (CALD), a
devastating cerebral demyelinating disease caused by loss of ABCD1 gene function. The …

Oxygen-glucose deprivation/reoxygenation-induced barrier disruption at the human blood–brain barrier is partially mediated through the HIF-1 pathway

S Page, S Raut, A Al-Ahmad - Neuromolecular medicine, 2019 - Springer
The blood–brain barrier (BBB) plays an important role in brain homeostasis.
Hypoxia/ischemia constitutes an important stress factor involved in several neurological …

[HTML][HTML] Trans-2-enoyl-CoA reductase Tecr-driven lipid metabolism in endothelial cells protects against transcytosis to maintain blood-brain barrier homeostasis

J Wang, J Xu, G Zang, T Zhang, Q Wu, H Zhang… - Research, 2022 - spj.science.org
The transport and metabolism of lipids in cerebrovascular endothelial cells (ECs) have been
hypothesized to regulate blood-brain barrier (BBB) maturation and homeostasis. Long-chain …

Chimerism in the realm of hematopoietic stem cell transplantation for non-malignant disorders—a perspective

C Zimmerman, S Shenoy - Frontiers in immunology, 2020 - frontiersin.org
Hematopoietic stem cell transplantation (HCT) is a curative intervention in non-malignant
disorders (NMD) that benefit from donor-derived hematopoiesis, immunity, and …

Evaluation of neurofilament light chain as a biomarker of neurodegeneration in X-linked childhood cerebral adrenoleukodystrophy

H Wang, MD Davison, ML Kramer, W Qiu… - Cells, 2022 - mdpi.com
Cerebral adrenoleukodystrophy (CALD) is a devastating, demyelinating neuroinflammatory
manifestation found in up to 40% of young males with an inherited mutation in ABCD1, the …

Neurocognitive benchmarks following transplant for emerging cerebral adrenoleukodystrophy

EI Pierpont, DR Nascene, R Shanley… - Neurology, 2020 - AAN Enterprises
Objective To quantify benchmark treatment outcomes that may be enabled by newborn
screening surveillance for X-linked adrenoleukodystrophy (ALD), we report neurocognitive …

The pathology of X-linked adrenoleukodystrophy: tissue specific changes as a clue to pathophysiology

HAF Yska, M Engelen, M Bugiani - Orphanet Journal of Rare Diseases, 2024 - Springer
Although the pathology of X-linked adrenoleukodystrophy (ALD) is well described, it
represents the end-stage of neurodegeneration. It is still unclear what cell types are initially …