Molecular BiologyMolecular Therapy — Nucleic AcidsPeer-reviewed
Effective Gene Therapy for Metachromatic Leukodystrophy Achieved with Minimal Lentiviral Genomic Integrations
Lucas Tricoli, Sunetra Sase, Julia Liao Hacker…·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.
3 citations