Article

Effective Gene Therapy for Metachromatic Leukodystrophy Achieved with Minimal Lentiviral Genomic Integrations

2024-03-14

Abstract excerpt

<h4>ABSTRACT</h4> Metachromatic leukodystrophy (MLD) is a fatal lysosomal storage disease (LSD) characterized by the deficient enzymatic activity of arylsulfatase A (ARSA). Combined autologous hematopoietic stem cell transplant (HSCT) with lentiviral (LV) based gene therapy has great potential to treat MLD. However, if enzyme production is inadequate, this could result in continued loss of motor function, implyin...

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Literature Corpus work: c1d618f1-1379-54c2-853e-98d1339b8c99
DOI: 10.1101/2024.03.14.584404

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