Durable multitransgene expression in vivo using systemic, nonviral DNA delivery

Recombinant adeno-associated virus (AAV) vectors are transforming therapies for rare human monogenic deficiency diseases. However, adaptive immune responses to AAV and its limited DNA insert capacity, restrict their therapeutic potential. HEDGES (high-level extended duration gene expression system),...

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Detalles Bibliográficos
Autores principales: Handumrongkul, Chakkrapong, Ye, Alice L., Chmura, Stephen A., Soroceanu, Liliana, Mack, Marissa, Ice, Ryan J., Thistle, Robert, Myers, Methawee, Ursu, Sarah J., Liu, Yong, Kashani-Sabet, Mohammed, Heath, Timothy D., Liggitt, Denny, Lewis, David B., Debs, Robert
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2019
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6881169/
https://www.ncbi.nlm.nih.gov/pubmed/31807699
http://dx.doi.org/10.1126/sciadv.aax0217

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