Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations

Metachromatic leukodystrophy (MLD) is a fatal lysosomal storage disease characterized by the deficient enzymatic activity of arylsulfatase A (ARSA). Combined autologous hematopoietic stem cell transplantion (HSCT) with lentiviral (LV)-based gene therapy has great potential to treat MLD. Achieving th...

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Autori principali:	Lucas Tricoli, Sunetra Sase, Julia L. Hacker, Vi Pham, Maxwell Chappell, Laura Breda, Stephanie N. Hurwitz, Naoto Tanaka, Carlo Castruccio Castracani, Amaliris Guerra, Zhongqi Hou, Lars Schlotawa, Karthikeyan Radhakrishnan, Matthew Hogenauer, Aoife Roche, John Everett, Frederic Bushman, Peter Kurre, Rebecca Ahrens-Nicklas, Laura A. Adang, Adeline L. Vanderver, Stefano Rivella
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	Elsevier 2025-03-01
Serie:	Molecular Therapy: Nucleic Acids
Soggetti:	MT: Delivery Strategies gene therapy gene complementation novel lentiviral vector metachromatic leukodystrophy MLD
Accesso online:	http://www.sciencedirect.com/science/article/pii/S2162253125000186
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