Enzyme-Loaded Gel Core Nanostructured Lipid Carriers to Improve Treatment of Lysosomal Storage Diseases: Formulation and In Vitro Cellular Studies of Elosulfase Alfa-Loaded Systems

• Main Authors: J. Víctor Álvarez (Author), Carolina Herrero Filgueira (Author), Alexandre de la Fuente González (Author), Cristóbal Colón Mejeras (Author), Andrés Beiras Iglesias (Author), Shunji Tomatsu (Author), José Blanco Méndez (Author), Asteria Luzardo Álvarez (Author), María Luz Couce (Author), Francisco J. Otero Espinar (Author)
• Format: Book
• Published: MDPI AG, 2019-10-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Mucopolysaccharidosis IVA (Morquio A) is a rare inherited metabolic disease caused by deficiency of the lysosomal enzyme N-acetylgalatosamine-6-sulfate-sulfatase (GALNS). Until now, treatments employed included hematopoietic stem cell transplantation and enzyme replacement therapy (ERT); the latter...