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PE7 prime editor

Development stage
Preclinical
Lead developer
Broad Institute
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

PE7 is an advanced CRISPR-based prime editing system designed to enhance genome editing efficiency by stabilizing prime editing guide RNAs (pegRNAs). It consists of a Cas9 nickase fused to a reverse transcriptase (RT), further modified with the N-terminal domain (amino acids 1-194) of the human La protein. The La protein component acts as an exonuclease protection factor, binding to the polyuridine tracts at the 3' ends of pegRNAs to prevent their degradation. Developed through research published in 2024, PE7 has demonstrated significantly higher editing rates compared to previous iterations like PEmax, particularly in challenging cell types such as primary human T cells and hematopoietic stem and progenitor cells (HSPCs). It is being investigated for the treatment of genetic disorders, including X-linked agammaglobulinemia (XLA), by enabling precise corrections of pathogenic variants without inducing double-strand DNA breaks.

Other names
PE7 prime editorPE-7 prime editorPE 7 prime editor
02

Targets

DNASSB (Lupus La antigen)NS5B (Hepatitis C virus non-structural protein 5B (NS5B) RNA-dependent RNA polymerase)

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