TY  - JOUR
AU  - Bertolin, Joan
AU  - Sánchez, Víctor
AU  - Ribera, Albert
AU  - Jaén, Maria Luisa
AU  - Garcia, Miquel
AU  - Pujol, Anna
AU  - Sánchez, Xavier
AU  - Muñoz, Sergio
AU  - Marcó, Sara
AU  - Pérez, Jennifer
AU  - Elias, Gemma
AU  - León, Xavier
AU  - Roca, Carles
AU  - Jimenez, Veronica
AU  - Otaegui, Pedro
AU  - Navarro, Marc
AU  - Ruberte, Jesús
AU  - Bosch, Fatima
AU  - Mulero, Francisca
PY  - 2021
DO  - 10.1038/s41467-021-25697-y
UR  - http://hdl.handle.net/20.500.12105/13728
AB  - Mucopolysaccharidosis type IVA (MPSIVA) or Morquio A disease, a lysosomal storage disorder, is caused by N-acetylgalactosamine-6-sulfate sulfatase (GALNS) deficiency, resulting in keratan sulfate (KS) and chondroitin-6-sulfate accumulation. Patients...
LA  - eng
PB  - Nature Publishing Group
KW  - ENZYME-REPLACEMENT THERAPY
KW  - MOUSE MODEL
KW  - N-ACETYLGALACTOSAMINE-6-SULFATE SULFATASE
KW  - ELOSULFASE ALPHA
KW  - KERATAN SULFATE
KW  - DRUG DELIVERY
KW  - MURINE MODEL
KW  - MPS IVA
KW  - MORQUIO
KW  - LIVER
KW  - Disease Models, Animal
KW  - Animals
KW  - Cartilage, Articular
KW  - Chondroitinsulfatases
KW  - Dependovirus
KW  - Gene Expression Regulation, Enzymologic
KW  - Genetic Therapy
KW  - Genetic Vectors
KW  - Humans
KW  - Male
KW  - Microscopy, Electron, Transmission
KW  - Mucopolysaccharidosis IV
KW  - Musculoskeletal System
KW  - Rats, Sprague-Dawley
KW  - Reverse Transcriptase Polymerase Chain Reaction
KW  - Treatment Outcome
TI  - Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy.
TY  - journal article
ER  -