Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy
Brief Summary: This is a phase I/II protocol aiming at the assessment of the safety and efficacy of ARSA gene transfer into hematopoietic stem/progenitor cells for the treatment of Metachromatic Leukodystrophy.