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Single, off-the-shelf treatment of Neuropathic Mucopolysaccharidoses with iPSC-derived microglia [bulk RNA-seq]

ABSTRACT: Single, off-the-shelf treatment of Neuropathic Mucopolysaccharidoses with iPSC-derived microglia [bulk RNA-seq]

PROVIDER: PRJNA924312 | ENA |

REPOSITORIES: ENA

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			Action	DRS
	SRR23092736.fastq.gz	Fastqsanger.gz
	SRR23092737.fastq.gz	Fastqsanger.gz
	SRR28817496.fastq.gz	Fastqsanger.gz
	SRR28817497.fastq.gz	Fastqsanger.gz
	SRR28817498.fastq.gz	Fastqsanger.gz

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Single, off-the-shelf treatment of Neuropathic Mucopolysaccharidoses with iPSC-derived microglia

Project description:Single, off-the-shelf treatment of Neuropathic Mucopolysaccharidoses with iPSC-derived microglia

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Single, off-the-shelf treatment of Neuropathic Mucopolysaccharidoses with iPSC-derived microglia [bulk RNA-seq]

Project description:Lysosomal storage disorders (LSDs) are a group of inherited metabolic disorders . A subgroup of LSDs is called Mucopolysaccharidoses (MPS), where accumulation of glycosaminoglycans causes a progressive degenerative disorder. The central nervous system (CNS) is particularly impacted with developmental delays, neurological regression, and early mortality. Current treatments are often insufficient to fully address the clinical need. Here we report the use of microglia derived from healthy human induced pluripotent stem cells (hiPSCs) as a potential one-time, allogeneic off-the-shelf cell therapy for several MPS. iPSC-derived microglia, termed MG01 , are replete with wild-type levels of lysosomal enzymes and we show that MG01 can deliver the functional enzyme into four different MPS knockout cell lines via mannose-6-phosphate receptor-mediated endocytosis in vitro. We then show that a single administration of MG01 provides sufficient enzyme to prevent behavioral deficits in two different animal models of MPS, efficacious to at least eight months after transplantation.

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Single, off-the-shelf treatment of Neuropathic Mucopolysaccharidoses with iPSC-derived microglia [scRNA-seq]

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