Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA

Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA

Abstract Objective Mucopolysaccharidosis type IIIA (MPSIIIA) caused by recessive SGSH variants results in sulfamidase deficiency, leading to neurocognitive decline and death. No disease‐modifying therapy is available. The AAVance gene therapy trial investigates AAVrh.10 overexpressing human sulfamid...

Full description

Bibliographic Details
Main Authors: Marianna Bugiani, Truus E. M. Abbink, Arthur W. D. Edridge, Lia van derHoek, Anne E. J. Hillen, Niek P. vanTil, Gino V. Hu‐A‐Ng, Marjolein Breur, Karen Aiach, Philippe Drevot, Michaël Hocquemiller, Ralph Laufer, Frits A. Wijburg, Marjo S. van derKnaap
Format: Article
Language:English
Published: Wiley 2023-06-01
Series:Annals of Clinical and Translational Neurology
Online Access:https://doi.org/10.1002/acn3.51772

Internet

https://doi.org/10.1002/acn3.51772

Similar Items