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Enhanced Efficacy from Gene Therapy in Pompe Disease Using Coreceptor Blockade
Enzyme replacement therapy (ERT) is the standard-of-care treatment of Pompe disease, a lysosomal storage disorder caused by deficiency of acid α-glucosidase (GAA). One limitation of ERT with recombinant human (rh) GAA is antibody formation against GAA. Similarly, in adeno-associated virus (AAV) vect...
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| I publikationen: | Hum Gene Ther |
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| Huvudupphovsmän: | , , , , , , , , , |
| Materialtyp: | Artigo |
| Språk: | Inglês |
| Publicerad: |
Mary Ann Liebert, Inc.
2015
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| Ämnen: | |
| Länkar: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4303018/ https://ncbi.nlm.nih.gov/pubmed/25382056 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2014.115 |
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