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A phase I-II, study of autologous cd34+ haematopoietic stem cells transduced ex vivo with cd11b lentiviral vector encoding for human SGSH in patients with mucopolysaccharidosis type IIIa (MPA IIIa, Sanfilippo Syndrome Type A).

Project Details

StatusActive
Effective start/end date1/10/24 → 30/06/27

Funding

  • University of Manchester: £26,820.00