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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Vydáno v:Hum Gene Ther
Hlavní autoři: Han, Sang-oh, Li, Songtao, Brooks, Elizabeth D., Masat, Elisa, Leborgne, Christian, Banugaria, Suhrad, Bird, Andrew, Mingozzi, Federico, Waldmann, Herman, Koeberl, Dwight
Médium: Artigo
Jazyk:Inglês
Vydáno: Mary Ann Liebert, Inc. 2015
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Research Articles
On-line přístup: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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