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- W3187112087 abstract "Recent advances in molecular biology have led to the CRISPR revolution, but the lack of an efficient and safe delivery system into cells and tissues continues to hinder clinical translation of CRISPR approaches. Polymeric vectors offer an attractive alternative to viruses as delivery vectors due to their large packaging capacity and safety profile. In this paper, we have demonstrated the potential use of a highly branched poly(β-amino ester) polymer, HPAE-EB, to enable genomic editing via CRISPRCas9-targeted genomic excision of exon 80 in the COL7A1 gene, through a dual-guide RNA sequence system. The biophysical properties of HPAE-EB were screened in a human embryonic 293 cell line (HEK293), to elucidate optimal conditions for efficient and cytocompatible delivery of a DNA construct encoding Cas9 along with two RNA guides, obtaining 15-20% target genomic excision. When translated to human recessive dystrophic epidermolysis bullosa (RDEB) keratinocytes, transfection efficiency and targeted genomic excision dropped. However, upon delivery of CRISPR-Cas9 as a ribonucleoprotein complex, targeted genomic deletion of exon 80 was increased to over 40%. Our study provides renewed perspective for the further development of polymer delivery systems for application in the gene editing field in general, and specifically for the treatment of RDEB." @default.
- W3187112087 created "2021-08-16" @default.
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- W3187112087 date "2021-08-06" @default.
- W3187112087 modified "2023-10-16" @default.
- W3187112087 title "Non-viral delivery of CRISPR–Cas9 complexes for targeted gene editing via a polymer delivery system" @default.
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- W3187112087 doi "https://doi.org/10.1038/s41434-021-00282-6" @default.
- W3187112087 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/34363036" @default.
- W3187112087 hasPublicationYear "2021" @default.
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