Children's Hospital of Philadelphia (CHOP) and the Perelman

The approach leverages microglial replacement in Krabbe disease to demonstrate its broader therapeutic potential across other neurological conditions.

In this study, researchers used a preclinical model to directly insert therapeutic cells in the brain, replacing GLD-associated microglia. They also created a large single-cell sequencing (scSeq) atlas of microglia before and after symptoms appeared, with and without replacement for comparison.

In the model with GLD, researchers observed relatively normal microglial maturation, followed by an early immune response which progressed to significant disruption of the normal functioning and regulation of microglia. The researchers noted a prominent transcriptional signature found in damaged white matter along with a molecular signature of globoid cells (GC).

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