Base Editors

Extracted Attributes

• Classes

  Cytosine Base Editors (CBEs), Adenine Base Editors (ABEs)

• Mechanism

  Combines CRISPR/Cas system with a deaminase enzyme for precise single-base substitution

• Advantages

  More precise and secure genome-editing effects than conventional artificial nuclease systems like CRISPR/Cas9, as it avoids severe genome damage from DSBs

• Key Feature

  Enables precise single-base substitution in DNA or RNA without generating DNA double-strand breaks (DSBs)

• Applications

  Biomedicine, gene function investigation, directed protein evolution, genetic lineage tracing, disease modeling, gene therapy

• Technology Type

  Genome editing, Gene editing, Genetic engineering

Timeline

• Publication of 'Base editing: advances and therapeutic opportunities' in Nature, detailing the unique mechanism of base editors in avoiding nucleic acid backbone cleavage. (Source: Web Search Results (Nature))

  2020-02-24

• Publication of 'Base editors: development and applications in biomedicine' in PubMed, reviewing recent developments and applications in the biomedical field. (Source: Web Search Results (PubMed))

  2023-07-11

• A scientific breakthrough occurred where advanced Base Editors were successfully used by physician Rebecca Arens Nicholas and researchers from UCSF to treat a fatal genetic disease in a baby, demonstrating the technology's therapeutic potential. (Source: Summary, Related Documents)

  Recent

Genome editing

Genome editing, or genome engineering, or gene editing, is a type of genetic engineering in which DNA is inserted, deleted, modified or replaced in the genome of a living organism. Unlike early genetic engineering techniques that randomly insert genetic material into a host genome, genome editing targets the insertions to site-specific locations. The basic mechanism involved in genetic manipulations through programmable nucleases is the recognition of target genomic loci and binding of effector DNA-binding domain (DBD), double-strand breaks (DSBs) in target DNA by the restriction endonucleases (FokI and Cas), and the repair of DSBs through homology-directed recombination (HDR) or non-homologous end joining (NHEJ).

Web Search Results

• Base editors: development and applications in biomedicine - PubMed

  Base editor (BE) is a gene-editing tool developed by combining the CRISPR/Cas system with an individual deaminase, enabling precise single-base substitution in DNA or RNA without generating a DNA double-strand break (DSB) or requiring donor DNA templates in living cells. Base editors offer more precise and secure genome-editing effects than other conventional artificial nuclease systems, such as CRISPR/Cas9, as the DSB induced by Cas9 will cause severe damage to the genome. Thus, base editors [...] have important applications in the field of biomedicine, including gene function investigation, directed protein evolution, genetic lineage tracing, disease modeling, and gene therapy. Since the development of the two main base editors, cytosine base editors (CBEs) and adenine base editors (ABEs), scientists have developed more than 100 optimized base editors with improved editing efficiency, precision, specificity, targeting scope, and capacity to be delivered in vivo, greatly enhancing their [...] application potential in biomedicine. Here, we review the recent development of base editors, summarize their applications in the biomedical field, and discuss future perspectives and challenges for therapeutic applications.

• CRISPR 101: Cytosine and Adenine Base Editors - Addgene Blog

  Base editors chemically modify DNA bases without breaking the DNA backbone, sidestepping the problem of HDR vs. NHEJ entirely. The first two classes of base editors were cytosine base editors (CBEs) and adenine base editors (ABEs). CBEs mediate a C to T change (or a G to A change on the opposite strand). ABEs make an A to G change (or a T to C change on the opposite strand). Both are base transition editors: converting one purine base to the other or one pyrimidine base to the other. [...] | A cartoon depiction of cytidine base editing. A base editor, consisting of a cytidine deaminase fused to Cas9 and UGI, is shown binding to DNA using its guide RNA. The guide RNA base pairs to target DNA, leaving the opposite strand of DNA free to be contacted by the cytidine deaminase, which converts a C to a U within this single-stranded sequence. This deamination yields DNA with a G:U mismatch without creating a double-strand break. Mismatch repair preserves the edit IF the modified strand [...] A cartoon depiction of cytidine base editing. A base editor, consisting of a cytidine deaminase fused to Cas9 and UGI, is shown binding to DNA using its guide RNA. The guide RNA base pairs to target DNA, leaving the opposite strand of DNA free to be contacted by the cytidine deaminase, which converts a C to a U within this single-stranded sequence. This deamination yields DNA with a G:U mismatch without creating a double-strand break. Mismatch repair preserves the edit IF the modified strand is

• Explainer: What Are Base Editors and How Do They Work?

  Base editing combines the powerful DNA-scanning and sequence-identification capabilities of the CRISPR-Cas9 system with a deaminase enzyme.

• Base editing: What is it and what does it mean for healthcare?

  Base editing is a type of genome editing – a way to make targeted changes to the sequence of a piece of DNA.

• Base editing: advances and therapeutic opportunities - Nature

  Base editing is unique in that it avoids nucleic acid backbone cleavage and, instead, directly chemically modifies target nucleobases in the