Evidence map›Paper›PMID 28957631›Full record

ReviewACS chemical biology2018

Editing the Genome Without Double-Stranded DNA Breaks.

Alexis C Komor, Ahmed H Badran, David R Liu

Registry-linked trialAbstract readReview
In one paragraph

Review in ACS chemical biology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06325709 (Phase 1/2 Trial of Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-linked Chronic Granulomatous Disease), which is not on this map. Cited by 70 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
70citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT06325709 phase1 / phase2recruitingnot on this mapstarted 2024, after this paper: background citation

Phase 1/2 Trial of Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-linked Chronic Granulomatous Disease

TypeinterventionalSponsorNational Institute of Allergy and Infectious Diseases (NIAID)Ran2024 to 2032Enrolled15ConditionsChronic Granulomatous Disease (CGD), X-Linked Chronic Granulomatous DiseaseArmsCampath, Sirolimus, Base-edited hematopoietic stem and progenitor cells, Busulfan, Palifermin
3 · Its place in the literature

Who cites it

70 citing papers in PubMed, 1 synthesis or guideline pooled it.

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10 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

3 authors.

Alexis C KomorDepartment of Chemistry and Biochemistry, University of California, San Diego , La Jolla, California 92093, United States.
Ahmed H BadranBroad Institute of MIT and Harvard , Cambridge, Massachusetts 021413, United States.
David R LiuBroad Institute of MIT and Harvard , Cambridge, Massachusetts 021413, United States.ORCID 0000-0002-9943-7557

Funding

Center for Genomic Editing and Recording: Development and Application of Next-Generation Genome and Epigenome Editing Methods to Advance the Study and Treatment of Human DiseaseRM1HG009490 · NHGRI · WHITEHEAD INSTITUTE FOR BIOMEDICAL RES · PI Brittany S. Adamson, Martin Joseph Ankrah Aryee · 2017 to 2026
$22.7M
Integrating Chemistry and Evolution to Illuminate Biology and Enable Novel TherapeuticsR35GM118062 · NIGMS · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2025
$6.4M
Continuous Evolution of Proteins with Novel Therapeutic PotentialR01EB022376 · NIBIB · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2019
$1.8M
Howard Hughes Medical InstituteNHGRI NIH HHS RM1 HG009490NIBIB NIH HHS R01 EB022376NIGMS NIH HHS R35 GM118062
6 · The paper itself

Abstract

Genome editing methods have commonly relied on the initial introduction of double-stranded DNA breaks (DSBs), resulting in stochastic insertions, deletions, and translocations at the target genomic locus. To achieve gene correction, these methods typically require the introduction of exogenous DNA repair templates and low-efficiency homologous recombination processes. In this review, we describe alternative, mechanistically motivated strategies to perform chemistry on the genome of unmodified cells without introducing DSBs. One such strategy, base editing, uses chemical and biological insights to directly and permanently convert one target base pair to another. Despite its recent introduction, base editing has already enabled a number of new capabilities and applications in the genome editing community. We summarize these advances here and discuss the new possibilities that this method has unveiled, concluding with a brief analysis of future prospects for genome and transcriptome editing without double-stranded DNA cleavage.

Indexed as

DNA Breaks, Double-StrandedGenomeAnimalsBacteriaCRISPR-Associated ProteinsCRISPR-Cas SystemsCytidine DeaminaseDNAEndonucleasesGene EditingMutagenesis, Site-DirectedPlantsRNA, Guide, CRISPR-Cas SystemsUracil-DNA GlycosidaseCRISPR-Associated ProteinsCytidine DeaminaseDNAEndonucleasesRNA, Guide, CRISPR-Cas SystemsUracil-DNA Glycosidase

Identifiers

PMID28957631
PMCPMC5891729

What OpenQuestion holds

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.