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Article

Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy.

Stem cells translational medicine - 1 Feb 2017

Meneghini Vasco, Frati Giacomo, Sala Davide, De Cicco Silvia, Luciani Marco, Cavazzin Chiara, Paulis Marianna, Mentzen Wieslawa, Morena Francesco, Giannelli Serena, Sanvito Francesca, Villa Anna, Bulfone Alessandro, Broccoli Vania, Martino Sabata, Gritti Angela

Abstract excerpt

Allogeneic fetal-derived human neural stem cells (hfNSCs) that are under clinical evaluation for several neurodegenerative diseases display a favorable safety profile, but require immunosuppression upon transplantation in patients. Neural progenitors derived from patient-specific induced pluripotent stem cells (iPSCs) may be relevant for autologous ex vivo gene-therapy applications to treat genetic diseases with...

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Identifiers

PMID: 28191778
DOI: 10.5966/sctm.2015-0414
PMCID: PMC5442804

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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