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Article

Neural stem cell engraftment and myelination in the human brain.

Science translational medicine - 10 Oct 2012

Gupta Nalin, Henry Roland G, Strober Jonathan, Kang Sang-Mo, Lim Daniel A, Bucci Monica, Caverzasi Eduardo, Gaetano Laura, Mandelli Maria Luisa, Ryan Tamara, Perry Rachel, Farrell Jody, Jeremy Rita J, Ulman Mary, Huhn Stephen L, Barkovich A James, Rowitch David H

Abstract excerpt

Pelizaeus-Merzbacher disease (PMD) is a rare leukodystrophy caused by mutation of the proteolipid protein 1 gene. Defective oligodendrocytes in PMD fail to myelinate axons, causing global neurological dysfunction. Human central nervous system stem cells (HuCNS-SCs) can develop into oligodendrocytes and confer structurally normal myelin when transplanted into a hypomyelinating mouse model. A 1-year, open-label...

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Identifiers

PMID: 23052294
DOI: 10.1126/scitranslmed.3004373
PMCID: PMC3893824

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026. Manifest publication-graph-cohort-2026-09-05.1.

Source receipt: pubmed, retrieved 5 Sept 2026.

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