Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease

Enzyme replacement therapy (ERT) is the current standard treatment for Pompe disease, a lysosomal storage disorder caused by deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). ERT has shown to be lifesaving in patients with classic infantile Pompe disease. However, a major drawback is...

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Main Authors: Qiushi Liang, Eva C. Vlaar, Fabio Catalano, Joon M. Pijnenburg, Merel Stok, Yvette van Helsdingen, Arnold G. Vulto, Wendy W.J. Unger, Ans T. van der Ploeg, W.W.M. Pim Pijnappel, Niek P. van Til
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2022-06-01
Colecção:Molecular Therapy: Methods & Clinical Development
Assuntos:
lentiviral gene therapy
Pompe disease
acid alpha-glucosidase
immune tolerance induction
antibody formation
immune response
Acesso em linha:http://www.sciencedirect.com/science/article/pii/S232905012200064X
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