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...

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Detalles Bibliográficos
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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Federation of American Societies for Experimental Biology 2015
Materias:
Research Communication
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

Internet

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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