Effective PEI-mediated delivery of CRISPR-Cas9 complex for targeted gene therapy.
Nanomedicine
; 14(7): 2095-2102, 2018 10.
Article
en En
| MEDLINE
| ID: mdl-29969727
ABSTRACT
The-state-of-art CRISPR/Cas9 is one of the most powerful among the approaches being developed to rescue fundamental causes of gene-based inheritable diseases. Several strategies for delivering such genome editing materials have been developed, but the safety, efficacy over time, cost of production, and gene size limitations are still under debate and must be addressed to further improve applications. In this study, we evaluated branched forms of the polyethylenimine (PEI) - branched PEI 25 kDa (BPEI-25K) - and found that it could efficiently deliver CRISPR/Cas9 plasmids. Plasmid DNA expressing both guide RNA and Cas9 to target the Slc26a4 locus was successfully delivered into Neuro2a cells and meditated genome editing within the targeted locus. Our results demonstrated that BPEI-25K is a promising non-viral vector to deliver the CRISPR/Cas9 system in vitro to mediate targeted gene therapy, and these findings contribute to an understanding of CRISPR/Cas9 delivery that may enable development of successful in vivo techniques.
Palabras clave
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Plásmidos
/
Polietileneimina
/
Terapia Genética
/
Sistemas de Liberación de Medicamentos
/
Sistemas CRISPR-Cas
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Transportadores de Sulfato
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Neuroblastoma
Límite:
Animals
Idioma:
En
Revista:
Nanomedicine
Asunto de la revista:
BIOTECNOLOGIA
Año:
2018
Tipo del documento:
Article