Highly efficient CRISPR-mediated large DNA docking and multiplexed prime editing using a single baculovirus.
Francesco AulicinoMartin PelosseChristine ToelzerJulien CapinErwin IlegemsParisa MeysamiRuth RollarsonPer-Olof BerggrenMark Simon DillinghamChristiane SchaffitzelMoin A SaleemGavin I WelshImre BergerPublished in: Nucleic acids research (2022)
CRISPR-based precise gene-editing requires simultaneous delivery of multiple components into living cells, rapidly exceeding the cargo capacity of traditional viral vector systems. This challenge represents a major roadblock to genome engineering applications. Here we exploit the unmatched heterologous DNA cargo capacity of baculovirus to resolve this bottleneck in human cells. By encoding Cas9, sgRNA and Donor DNAs on a single, rapidly assembled baculoviral vector, we achieve with up to 30% efficacy whole-exon replacement in the intronic β-actin (ACTB) locus, including site-specific docking of very large DNA payloads. We use our approach to rescue wild-type podocin expression in steroid-resistant nephrotic syndrome (SRNS) patient derived podocytes. We demonstrate single baculovirus vectored delivery of single and multiplexed prime-editing toolkits, achieving up to 100% cleavage-free DNA search-and-replace interventions without detectable indels. Taken together, we provide a versatile delivery platform for single base to multi-gene level genome interventions, addressing the currently unmet need for a powerful delivery system accommodating current and future CRISPR technologies without the burden of limited cargo capacity.
Keyphrases
- crispr cas
- genome editing
- genome wide
- single molecule
- circulating tumor
- living cells
- cell free
- highly efficient
- molecular dynamics
- wild type
- molecular dynamics simulations
- poor prognosis
- sars cov
- nucleic acid
- fluorescent probe
- circulating tumor cells
- single cell
- dna methylation
- copy number
- high throughput
- transcription factor
- current status
- risk factors
- diabetic nephropathy
- genome wide analysis