GENE THERAPY IN MUCOPOLYSACCHARIDOSIS TYPE IIIA: CASE REPORTS

• Main Authors: Benjamin Lah (Author), Tadej Jalšovec (Author), Ana Drole Torkar (Author), Jana Kodrič (Author), Saba Battelino (Author), Mojca Žerjav Tanšek (Author), Tadej Battelino (Author), Urh Grošelj (Author)
• Format: Book
• Published: The Society for Children with Metabolic Disorders, 2022-05-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Mucopolysaccharidoses are a group of rare lysosomal storage diseases. The clinical signs develop gradually, the impairment is progressive and multiple organs are affected. With the currently known treatment options, the patient cannot be cured. However, with the arrival of effective gene therapy, to...