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Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates
Several acute monogenic diseases affect multiple body systems, causing death in childhood. The development of novel therapies for such conditions is challenging. However, improvements in gene delivery technology mean that gene therapy has the potential to treat such disorders. We evaluated the abili...
Autores principales: | Mattar, Citra N., Wong, Andrew M. S., Hoefer, Klemens, Alonso-Ferrero, Maria E., Buckley, Suzanne M. K., Howe, Steven J., Cooper, Jonathan D., Waddington, Simon N., Chan, Jerry K. Y., Rahim, Ahad A. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Federation of American Societies for Experimental Biology
2015
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4560173/ https://www.ncbi.nlm.nih.gov/pubmed/26062602 http://dx.doi.org/10.1096/fj.14-269092 |
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