Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates

Pompe disease (PD) is a severe neuromuscular disorder caused by deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). PD is currently treated with enzyme replacement therapy (ERT) with intravenous infusions of recombinant human GAA (rhGAA). Although the introduction of ERT represents a br...

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Detalles Bibliográficos
Autores principales: Costa-Verdera, Helena, Collaud, Fanny, Riling, Christopher R., Sellier, Pauline, Nordin, Jayme M. L., Preston, G. Michael, Cagin, Umut, Fabregue, Julien, Barral, Simon, Moya-Nilges, Maryse, Krijnse-Locker, Jacomina, van Wittenberghe, Laetitia, Daniele, Natalie, Gjata, Bernard, Cosette, Jeremie, Abad, Catalina, Simon-Sola, Marcelo, Charles, Severine, Li, Mathew, Crosariol, Marco, Antrilli, Tom, Quinn, William J., Gross, David A., Boyer, Olivier, Anguela, Xavier M., Armour, Sean M., Colella, Pasqualina, Ronzitti, Giuseppe, Mingozzi, Federico
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8568898/
https://www.ncbi.nlm.nih.gov/pubmed/34737297
http://dx.doi.org/10.1038/s41467-021-26744-4

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