Effective Gene Therapy for Metachromatic Leukodystrophy Achieved with Minimal Lentiviral Genomic Integrations
Lucas Tricoli, Sunetra Sase, Julia Liao 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 M. Roche, , Stefano Rivella
This paper develops a novel lentiviral vector for gene therapy of MLD that achieves high ARSA activity with minimal genomic integrations, improving safety and efficacy in preclinical models.
Molecular Therapy — Nucleic Acids · Jan 25, 2025Peer-reviewedMolecular Biology