Article

An innovative hematopoietic stem cell gene therapy approach benefits CLN1 disease in the mouse model

2022-03-04

Abstract excerpt

Hematopoietic stem and progenitor cells (HSPCs) can lead to the establishment of a long-lasting microglia-like progeny in the brain of properly myeloablated hosts. We exploited this approach to treat the severe CLN1 neurodegenerative disorder, which is the most aggressive form of neuronal ceroid lipofuscinoses, due to deficiency of palmitoyl-protein thioesterase 1 (hPPT1). We here provide first evidence that: i) t...

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Literature Corpus work: 233d5b3d-c460-5fe9-a597-37ddf341ef03
DOI: 10.1101/2022.03.03.482460

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