Prolonged Expression of Secreted Enzymes in Dogs After Liver-Directed Delivery of Sleeping Beauty Transposons: Implications for Non-Viral Gene Therapy of Systemic Disease

The non-viral, integrating Sleeping Beauty (SB) transposon system is efficient in treating systemic monogenic disease in mice, including hemophilia A and B caused by deficiency of blood clotting factors and mucopolysaccharidosis types I and VII caused by α-L-iduronidase (IDUA) and β-glucuronidase (G...

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Detaylı Bibliyografya
Yayımlandı:Hum Gene Ther
Asıl Yazarlar: Aronovich, Elena L., Hyland, Kendra A., Hall, Bryan C., Bell, Jason B., Olson, Erik R., Rusten, Myra Urness, Hunter, David W., Ellinwood, N. Matthew, McIvor, R. Scott, Hackett, Perry B.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Mary Ann Liebert, Inc. 2017
Konular:
Research Articles
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC5549796/
https://ncbi.nlm.nih.gov/pubmed/28530135
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2017.004
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