The article discusses the potential of lentiviral-based gene therapy for treating rare genetic diseases affecting the brain and spinal cord, including X-linked adrenoleukodystrophy, metachromatic leukodystrophy, and mucopolysaccharidoses. It highlights the aim of improving enzyme bioavailability and correcting neuropathological phenotypes.
The first clinical application of lentiviral-based gene therapy was for treatment of inherited metabolic disorders including X-linked adrenoleukodystrophy (Eichler et al., 2017), metachromatic leukodystrophy (Rosenberg et al., 2016), and mucopolysaccharidoses (Kinsella et al., 2020), with the aim of increasing enzyme bioavailability and brain entry (Begley et al., 2008) for correction of the neuropathological phenotype (see below in the disease sections for more details).
Original title: “Frontiers | Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord”