Direct Microglia Replacement Reveals Pathologic and Therapeutic Contributions of Brain Macrophages to a Monogenic Neurological Disease

Medicine, Neurology, Life Sciences, Cell Biology, Immunology,
microglia   macrophages   monogenic disease   neuropathology   therapy  

Academic Background Krabbe disease (also known as globoid cell leukodystrophy, GLD) is a fatal pediatric neurodegenerative disorder caused by mutations in the galactosylceramidase (GALC) gene. A hallmark pathological feature of this disease is the presence of lipid-laden globoid cells (GCs) in the central nervous system (CNS). Currently, hematopoie...

Persistent NRG1 Type III Overexpression in Spinal Motor Neurons Has No Therapeutic Effect on ALS-Related Pathology in SOD1 G93A Mice

Medicine, Basic Medical Sciences, Neurology, Life Sciences, Cell Biology, Biochemistry, Immunology, Microbiology,
ALS   motor neurons   NRG1   SOD1   neurodegenerative disease   therapy   gene therapy  

Background and Research Motivation Amyotrophic Lateral Sclerosis (ALS) is a devastating neurodegenerative disease affecting upper and lower motor neurons, leading to progressive muscle paralysis and eventual death. Currently, no effective treatments can significantly delay or halt ALS progression. While various ALS mouse models (such as those carry...