Dose-dependent effects of enzyme replacement therapy on skeletal disease progression in mucopolysaccharidosis VII dogs

Mucopolysaccharidosis (MPS) VII is an inherited lysosomal storage disorder characterized by deficient activity of the enzyme β-glucuronidase. Skeletal abnormalities are common in patients and result in diminished quality of life. Enzyme replacement therapy (ERT) for MPS VII using recombinant human β...

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Detalles Bibliográficos
Autores principales: Gawri, Rahul, Lau, Yian Khai, Lin, Gloria, Shetye, Snehal S., Zhang, Chenghao, Jiang, Zhirui, Abdoun, Khaled, Scanzello, Carla R., Jo, Stephanie Y., Mai, Wilfried, Dodge, George R., Casal, Margret L., Smith, Lachlan J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9747356/
https://www.ncbi.nlm.nih.gov/pubmed/36570425
http://dx.doi.org/10.1016/j.omtm.2022.11.006

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