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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...
Autores principales: | Doerfler, Phillip A, Nayak, Sushrusha, Corti, Manuela, Morel, Laurence, Herzog, Roland W, Byrne, Barry J |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2016
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Materias: | |
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 |
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