Inclusion Criteria:
* Documented MPS IIIA diagnosis based on genotyping confirming the SGSH gene mutations
* Cognitive DQ score on BSID-III: 50% and above
Exclusion Criteria:
* Homozygous for the S298P mutation or non-classical severe form of MPS IIIA, based on investigator's judgement.
* Participation in another gene or cell therapy clinical trial.
* Past use of SGSH enzyme replacement therapy for a period exceeding 3 months. A washout period of at least 2 months is required prior to screening.
* Current participation in a clinical trial of another investigational medicinal product.
* History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following surgery.
* Any condition that would contraindicate treatment with immunosuppressants such as tacrolimus, mycophenolate mofetil or steroids.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov