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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...
में बचाया:
| में प्रकाशित: | Mol Ther Methods Clin Dev |
|---|---|
| मुख्य लेखकों: | , , , , , |
| स्वरूप: | Artigo |
| भाषा: | Inglês |
| प्रकाशित: |
Nature Publishing Group
2016
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| विषय: | |
| ऑनलाइन पहुंच: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4729315/ https://ncbi.nlm.nih.gov/pubmed/26858964 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2015.53 |
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