Targeted approaches to induce immune tolerance for Pompe disease therapy

Enzyme and gene replacement strategies have developed into viable therapeutic approaches for the treatment of Pompe disease (acid α-glucosidase (GAA) deficiency). Unfortunately, the introduction of GAA and viral vectors encoding the enzyme can lead to detrimental immune responses that attenuate trea...

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Detalles Bibliográficos
Autores principales: Doerfler, Phillip A, Nayak, Sushrusha, Corti, Manuela, Morel, Laurence, Herzog, Roland W, Byrne, Barry J
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Review Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4729315/
https://www.ncbi.nlm.nih.gov/pubmed/26858964
http://dx.doi.org/10.1038/mtm.2015.53

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4729315/
https://www.ncbi.nlm.nih.gov/pubmed/26858964
http://dx.doi.org/10.1038/mtm.2015.53

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