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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Dades bibliogràfiques
Publicat a:Hum Gene Ther
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2017
Matèries:
Research Articles
Accés en línia: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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