Identifying altered developmental pathways in human globoid cell leukodystrophy ipscsderived nscs using transcriptome profiling

HIGHLIGHTS

• who: Neurodegenerative and colleagues from the Globoid cell leukodystrophy (GLD), also known as Krabbe disease, is a lysosomal storage disease that is triggered by a deficit of the lysosomal enzyme galactosylceramidase (GALC) and is characterized by psychosine (PSY) accumulation in the nervous system [1]A total of , mutations are known to cause GLD in the human GALC gene located on chromosome, q, [2-, ]. The absence of GALC results in the accumulation of PSY which has strong neurotoxic activity that can lead to extensive demyelination, neuroinflammation, and axonal degeneration of the nervous system [2]. In addition to acting . . .
  

   

  

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