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Based on these data, an ongoing phase 1/2a, multicenter, open-label, ascending dose, first in-human clinical trial is planned to evaluate the safety of intracisternal PR001A (AAV9-GBA1) administration in patients with moderate to severe PD with at least one GBA pathogenic variant (NCT04127578, Table 2)

Work involving the use of fluorescent substrates of Pgp and MRP as probes for the presence of these transporters discovered that ABC transporters are localized in the S