Nanodevices for the Efficient Codelivery of CRISPR-Cas9 Editing Machinery and an Entrapped Cargo: A Proposal for Dual Anti-Inflammatory Therapy.

Autores de IIS La Fe
Participantes ajenos a IIS La Fe
- Garcia-Fernandez, Alba
- Vivo-Llorca, Gema
- Sancho, Monica
- García-Jareño AB
- Ramirez-Jimenez, Laura
- Barber-Cano, Eloisa
- Murguía JR
- Orzaez, Mar
Grupos
Abstract
In this article, we report one of the few examples of nanoparticles capable of simultaneously delivering CRISPR-Cas9 gene-editing machinery and releasing drugs for one-shot treatments. Considering the complexity of inflammation in diseases, the synergistic effect of nanoparticles for gene-editing/drug therapy is evaluated in an in vitro inflammatory model as proof of concept. Mesoporous silica nanoparticles (MSNs), able to deliver the CRISPR/Cas9 machinery to edit gasdermin D (GSDMD), a key protein involved in inflammatory cell death, and the anti-inflammatory drug VX-765 ((CRISPR)-C-GSDMD45-VX-MSNs), were prepared. Nanoparticles allow high cargo loading and CRISPR-Cas9 plasmid protection and, thus, achieve the controlled codelivery of CRISPR-Cas9 and the drug in cells. Nanoparticles exhibit GSDMD gene editing by downregulating inflammatory cell death and achieving a combined effect on decreasing the inflammatory response by the codelivery of VX-765. Taken together, our results show the potential of MSNs as a versatile platform by allowing multiple combinations for gene editing and drug therapy to prepare advanced nanodevices to meet possible biomedical needs.
Datos de la publicación
- ISSN/ISSNe:
- 1999-4923, 1999-4923
- Tipo:
- Article
- Páginas:
- -
- PubMed:
- 35890389
- Factor de Impacto:
- 0,922 SCImago ℠
- Cuartil:
- Q1 SCImago ℠
Pharmaceutics Multidisciplinary Digital Publishing Institute (MDPI)
Citas Recibidas en Web of Science: 11
Documentos
- No hay documentos
Filiaciones
Keywords
- mesoporous silica nanoparticles; CRISPR-Cas9; gene editing; inflammation; drug delivery
Proyectos y Estudios Clínicos
NANOMATERIALES INTELIGENTES, SONDAS Y DISPOSITIVOS PARA EL DESARROLLO INTEGRADO DE NUEVAS HERRAMIENTAS APLICADAS AL CAMPO BIOMÉDICO
Investigador Principal: JUAN BAUTISTA SALOM SANVALERO
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Investigador Principal: RAMÓN MARTÍNEZ MÁÑEZ
PTA2016-12603-1 . MINISTERIO DE ECONOMIA Y COMPETITIVIDAD . 2018
PLATAFORMA PARA LA DETECCIÓN DE PATÓGENOS BASADA EN MATERIALES CON PUERTAS MOLECULARES (PATH-GATE).
Investigador Principal: RAMÓN MARTÍNEZ MÁÑEZ
DTS18/00090 . INSTITUTO DE SALUD CARLOS III . 2019
Contrato i-PFIS Doctorados IIS - empresa en Ciencias y Tecnologías de la Salud.
Investigador Principal: FÉLIX SANCENÓN GALARZA
IFI19/00026 . INSTITUTO DE SALUD CARLOS III . 2020
Contratos Sara Borrell 2020. DIEZ SÁNCHEZ, PAULA
Investigador Principal: FÉLIX SANCENÓN GALARZA
CD20/00120 . INSTITUTO DE SALUD CARLOS III . 2021
Cita
Garcia A,Vivo G,Sancho M,García AB,Ramirez L,Barber E,Murguía JR,Orzaez M,SANCENON F,MARTINEZ R. Nanodevices for the Efficient Codelivery of CRISPR-Cas9 Editing Machinery and an Entrapped Cargo: A Proposal for Dual Anti-Inflammatory Therapy. Pharmaceutics. 2022. 14. (7):1495. IF:5,400. (1).