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Improved split prime editors enable efficient in vivo genome editing [Amplicon_Seq_in_vitro]

ABSTRACT: Improved split prime editors enable efficient in vivo genome editing [Amplicon_Seq_in_vitro]

PROVIDER: PRJNA1128634 | ENA |

REPOSITORIES: ENA

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			Action	DRS
	SRR29590801_1.fastq.gz	Fastqsanger.gz
	SRR29590801_2.fastq.gz	Fastqsanger.gz
	SRR29590702_1.fastq.gz	Fastqsanger.gz
	SRR29590702_2.fastq.gz	Fastqsanger.gz
	SRR29590802_1.fastq.gz	Fastqsanger.gz

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Improved split prime editors enable efficient in vivo genome editing

Project description:Improved split prime editors enable efficient in vivo genome editing

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Improved split prime editors enable efficient in vivo genome editing

Project description:Prime editor (PE) is a precise genome-editing tool capable of all possible base conversions, as well as insertions and deletions without DSBs or donor DNA. The efficient delivery of PE in vivo is critical for realizing its full potential in disease modeling and therapeutic correction. Although PE has been divided into two halves and delivered using dual adeno-associated viruses (AAVs), editing efficiency at different gene loci varies among split sites, and efficient split sites within Cas9 nickase are limited. In this study, by screening multiple split sites, we demonstrated a series of efficient split site when delivering PE by dual-AAV. Additionally, we utilized a feature reported by others recently that RNase could be detached from the Cas9n and designed split sites in the first half of Cas9n. To test the editing efficiency in vivo, a novel dual-AAV split-ePE3 was packaged in AAV9 and delivered via tail vein injection in mice, achieving 24.4% precise genome editing 3 weeks post-injection. Our findings establish an alternative split-PE architecture that could achieve robust gene editing efficiency, facilitating the potential utility both in model organisms and as a therapeutic modality.

2024-12-01 | GSE270813 | GEO

Improved split prime editors enable efficient in vivo genome editing [Amplicon_Seq_in_vitro]

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Targeted exon skipping with AAV-mediated split adenine base editors.

Project description:Techniques for exclusion of exons from mature transcripts have been applied as gene therapies for treating many different diseases. Since exon skipping has been traditionally accomplished using technologies that have a transient effect, it is particularly important to develop new techniques that enable permanent exon skipping. We have recently demonstrated that this can be accomplished using cytidine base editors for permanently disabling the splice acceptor of target exons. We now demonstrate the application of adenine-deaminase base editors to disrupt the conserved adenosine within splice acceptor sites for programmable exon skipping. We also demonstrate that by altering the amino acid sequence of the linker between the adenosine deaminase domain and the Cas9 nickase or by coupling the adenine base editor with a uracil glycosylase inhibitor, the DNA editing efficiency and exon skipping rates improve significantly. Finally, we developed a split base editor architecture compatible with adeno-associated viral packaging. Collectively, these results represent significant progress towards permanent in vivo exon skipping through base editing and, ultimately, a new modality of gene therapy for the treatment of genetic diseases.

2019-09-12 | GSE120125 | GEO

Transcriptome wide edits after split-engineered base editor treatment

Project description:We report transcriptome wide edits comparison between split-engineered base editors and intact base editors. Our results show that, split-engineered base editors show backgound levels of unique C>U edits when compared to intact base editors.

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Targeted exon skipping with AAV-mediated split adenine base editors.

Project description:Targeted exon skipping with AAV-mediated split adenine base editors.

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Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice.

Project description:Prime editors (PEs) mediate genome modification without utilizing double-stranded DNA breaks or exogenous donor DNA as a template. PEs facilitate nucleotide substitutions or local insertions or deletions within the genome based on the template sequence encoded within the prime editing guide RNA (pegRNA). However, the efficacy of prime editing in adult mice has not been established. Here we report an NLS-optimized SpCas9-based prime editor that improves genome editing efficiency in both fluorescent reporter cells and at endogenous loci in cultured cell lines. Using this genome modification system, we could also seed tumor formation through somatic cell editing in the adult mouse. Finally, we successfully utilize dual adeno-associated virus (AAVs) for the delivery of a split-intein prime editor and demonstrate that this system enables the correction of a pathogenic mutation in the mouse liver. Our findings further establish the broad potential of this genome editing technology for the directed installation of sequence modifications in vivo, with important implications for disease modeling and correction.

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A truncated reverse transcriptase enhances prime editing by split AAV vectors

Project description:A truncated reverse transcriptase enhances prime editing by split AAV vectors

| PRJNA846287 | ENA

Highly efficient prime editing by introducing same-sense mutations in pegRNA or stabilizing its structure.

Project description:Prime editors (PEs) can mediate versatile genome editing but their efficiency remains low. Here, we developed spegRNA by introducing same-sense mutations at proper positions in the reverse-transcription template of pegRNA to increase PE’s single-base editing efficiency or apegRNA by altering the pegRNA secondary structure to increase PE’s indel-editing efficiency . When used in PE3 and PE5, the efficiencies of sPE3, aPE3, sPE5 and aPE5 were all enhanced significantly.

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A truncated reverse transcriptase enhances prime editing by split AAV vectors

Project description:To investigate if the truncated PE can be dilivered by dual AAV8 vectors for in vivo prime editing. We injected the dual AAV8 into 10-week-old C57BL/6J mice . Livers were isolated 4 weeks after injection and next generation sequencing showed an average of 1.4% and 5.4% precise prime editing with the low and high AAV doses, respectively (Figure 4D ). This demonstrates that PECO-Mini can be efficiently delivered by dual AAVs for in vivo prime editing.

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