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Predicting Gene Silencing Through the Spatiotemporal Control of siRNA Release from Photo-responsive Polymeric Nanocarriers
Published on: July 21, 2017
Polímero colgante:amino-β-ciclodextrina:siRNA huésped:nanopartículas huésped como vectores eficientes para el
Aditya Kulkarni1, Kyle DeFrees, Seok-Hee Hyun
1Department of Chemistry, Purdue University, 560 Oval Drive, West Lafayette, Indiana 47907, USA.
Journal of the American Chemical Society
|May 2, 2012
Resumen
Un nuevo sistema de autoensamblaje de nanopartículas ofrece efectivamente siRNA. Este nuevo vector, hecho de beta-ciclodextrina catiónica y un polímero degradable, muestra baja toxicidad y silenciamiento de genes comparable a los métodos existentes.
Área de la Ciencia:
- Ciencia de los Biomateriales Ciencia de los Biomateriales.
- Nanotecnología La nanotecnología es la nanotecnología.
- Entrega de genes Entrega de genes
Sus antecedentes:
- El desarrollo de vectores de entrega de siRNA seguros y efectivos es crucial para la terapia génica.
- Los vectores no virales existentes a menudo se enfrentan a desafíos de toxicidad y eficiencia.
- Los nanomateriales autoensambladores ofrecen una plataforma prometedora para la administración de fármacos y ácidos nucleicos dirigidos.
Objetivo del estudio:
- Desarrollar y caracterizar un nuevo sistema de autoensamblaje de nanopartículas para la entrega de siRNA.
- Evaluar la eficiencia y la citotoxicidad del vector desarrollado.
- Para comparar el rendimiento del nuevo vector con los reactivos de transfección de referencia.
Principales métodos:
- Síntesis de monosubstituidos derivados catiónicos de la beta-ciclodextrina.
- Preparación de poli (alcohol vinílico) (PVA) con injertos de poli (etilenglicol) (PEG) y colesterol (Chol) a través de un enlace ácido-lábil.
- Investigación de la formación de nanopartículas utilizando dos esquemas de autoensamblaje con siRNA.
- Caracterización del tamaño de las nanopartículas y el potencial zeta.
- Evaluación de la viabilidad celular y la eficiencia de silenciamiento de genes en las células CHO-GFP.
Principales resultados:
- El autoensamblaje de los derivados beta-CD catiónicos y las nanopartículas formadas por el polímero Chol-PVA-PEG (120-170 nm) con siRNA.
- Las nanopartículas exhibieron un potencial zeta ligeramente negativo.
- El nuevo vector demostró una citotoxicidad 1000 veces menor en comparación con 25 kDa bPEI.
- Las eficiencias de silenciamiento de genes fueron comparables a los reactivos de referencia como bPEI y Lipofectamina 2000.
Conclusiones:
- El sistema de autoensamblaje desarrollado forma efectivamente nanopartículas para la entrega de siRNA.
- El nuevo vector ofrece una alternativa de baja toxicidad para aplicaciones de silenciamiento de genes.
- Este enfoque tiene potencial para el avance de las terapias de siRNA no virales.
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