MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT
Status:
Not yet recruiting
Trial end date:
2023-06-01
Target enrollment:
Participant gender:
Summary
This is a single center, Phase 1/2 study in which patients with Hurler syndrome who have
previously undergone allogeneic hematopoietic stem cell transplantation are treated with
autologous plasmablasts engineered to express α-L-iduronidase (IDUA) using the Sleeping
Beauty transposon system.