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

Authors: 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

Source: Molecular Therapy: Nucleic Acids, Vol 36, Iss 1, Pp 102464- (2025)

Subject Terms: MT: Delivery Strategies, gene therapy, gene complementation, novel lentiviral vector, metachromatic leukodystrophy, MLD, Therapeutics. Pharmacology, RM1-950

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2162253125000186; https://doaj.org/toc/2162-2531

Access URL: https://doaj.org/article/b59f946719ad4f259d2bf5d4829f0c32