Evidence map›Paper›PMID 39212664›Full record

ArticleHuman gene therapy2024

Unconstrained Precision Mitochondrial Genome Editing with αDdCBEs.

Santiago R Castillo, Brandon W Simone, Karl J Clark, Patricia Devaux, Stephen C Ekker

Abstract read
In one paragraph

Article in Human gene therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
–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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

11 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
  4. Review
  5. Review
  6. Review
  7. Article
  8. Review
  9. Gene Therapy Techniques and Delivery Methods (Review).Sovremennye tekhnologii v meditsine · 2025
    Review
  10. Article
  11. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

5 authors.

Santiago R CastilloVirology and Gene Therapy Graduate Program, Mayo Clinic, Rochester, Minnesota, USA.ORCID 0000-0001-7881-1813
Brandon W SimoneDepartment of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota, USA.
Karl J ClarkDepartment of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota, USA.
Patricia DevauxVirology and Gene Therapy Graduate Program, Mayo Clinic, Rochester, Minnesota, USA.
Stephen C EkkerDepartment of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota, USA.

Funding

Systematic Vertebrate Functional GenomicsR01GM063904 · NIGMS · UNIVERSITY OF TEXAS AT AUSTIN · PI EKKER, STEPHEN CARL · 2001 to 2025
$10.7M
Conditional genetics and precision genome editing resources for ZebrafishR24OD036201 · OD · IOWA STATE UNIVERSITY · PI KARL J CLARK, Stephen Carl Ekker · 2024 to 2026
$3.0M
Building the mitochondrial genome editing repertoireU01AI142773 · NIAID · MAYO CLINIC ROCHESTER · PI CLARK, KARL J, EKKER, STEPHEN CARL · 2018 to 2022
$2.0M
NIAID NIH HHS U01 AI142773NIGMS NIH HHS R01 GM063904NIH HHS R24 OD036201
6 · The paper itself

Abstract

DddA-derived cytosine base editors (DdCBEs) enable the targeted introduction of C•G-to-T•A conversions in mitochondrial DNA (mtDNA). DdCBEs work in pairs, with each arm composed of a transcription activator-like effector (TALE), a split double-stranded DNA deaminase half, and a uracil glycosylase inhibitor. This pioneering technology has helped improve our understanding of cellular processes involving mtDNA and has paved the way for the development of models and therapies for genetic disorders caused by pathogenic mtDNA variants. Nonetheless, given the intrinsic properties of TALE proteins, several target sites in human mtDNA are predicted to remain out of reach to DdCBEs and other TALE-based technologies. Specifically, due to the conventional requirement for a thymine immediately upstream of the TALE target sequences (

Indexed as

DNA, MitochondrialGene EditingGenome, MitochondrialCRISPR-Cas SystemsCytosineHumansMitochondriaTranscription Activator-Like Effector NucleasesTranscription Activator-Like EffectorsCytosineDNA, MitochondrialTranscription Activator-Like Effector NucleasesTranscription Activator-Like EffectorsDddA-derived cytosine base editors (DdCBEs)mitochondrial base editingmitochondrial DNA (mtDNA)precision genome engineeringtranscription activator-like effectors (TALEs)

Identifiers

PMID39212664
PMCPMC11511777

What Socratic holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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 Socratic graph.