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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Detalles Bibliográficos
Autores principales: Liang, Qiushi, Vlaar, Eva C., Catalano, Fabio, Pijnenburg, Joon M., Stok, Merel, van Helsdingen, Yvette, Vulto, Arnold G., Unger, Wendy W.J., van der Ploeg, Ans T., Pijnappel, W.W.M. Pim, van Til, Niek P.
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/PMC9127119/
https://www.ncbi.nlm.nih.gov/pubmed/35662813
http://dx.doi.org/10.1016/j.omtm.2022.04.016

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9127119/
https://www.ncbi.nlm.nih.gov/pubmed/35662813
http://dx.doi.org/10.1016/j.omtm.2022.04.016

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