Adeno-associated virus-based gene therapy delivering combinations of two growth-associated genes to MPS IVA mice

• Main Authors: Estera Rintz (Author), Betul Celik (Author), Nidhi Fnu (Author), Angélica María Herreño-Pachón (Author), Shaukat Khan (Author), Eliana Benincore-Flórez (Author), Shunji Tomatsu (Author)
• Format: Book
• Published: Elsevier, 2024-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Mucopolysaccharidosis type IVA (MPS IVA) is caused by a deficiency of the galactosamine (N-acetyl)-6-sulfatase (GALNS) enzyme responsible for the degradation of specific glycosaminoglycans (GAGs). The progressive accumulation of GAGs leads to various skeletal abnormalities (short stature, hypoplasia...