Evidence map›Paper›PMID 34141821›Full record

ArticleMolecular therapy. Methods & clinical development2021

Targeting the

Marco De Giorgi, Ang Li, Ayrea Hurley, Mercedes Barzi, Alexandria M Doerfler, Nikitha A Cherayil, Harrison E Smith, Jonathan D Brown, Charles Y Lin, Karl-Dimiter Bissig and 2 more

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed
1.3field-weighted citation impact, top 20% of its field
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

16 citing papers in PubMed, 27 citations in OpenAlex.

  1. Article
  2. Cytosine base editing of LPA in transgenic mice averts large deletions.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  3. Article
  4. International journal of biological sciences · 2026
    Review
  5. Article
  6. Repair Drive improves gene editing in the liver.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  7. Review
  8. Article
  9. Article
  10. Review
  11. A promoterless AAV6.2FF-based lung gene editing platform for the correction of surfactant protein B deficiency.Molecular therapy : the journal of the American Society of Gene Therapy · 2023
    Article
  12. Article
  13. Review
  14. Review
  15. Article
  16. Article
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

12 authors at 4 institutions in 1 country.

Marco De GiorgiDepartment of Molecular Physiology and Biophysics, Baylor College of Medicine, Houston, TX 77030, USA.
Ang LiDepartment of Bioengineering, Rice University, Houston, TX 77030, USA.
Ayrea HurleyDepartment of Molecular Physiology and Biophysics, Baylor College of Medicine, Houston, TX 77030, USA.
Mercedes BarziDepartment of Pediatrics, Division of Medical Genetics, Duke University, Durham, NC 27710, USA.
Alexandria M DoerflerDepartment of Molecular Physiology and Biophysics, Baylor College of Medicine, Houston, TX 77030, USA.
Nikitha A CherayilDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
Harrison E SmithDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
Jonathan D BrownDivision of Cardiovascular Medicine, Vanderbilt University Medical Center, Nashville, TN 37232, USA.
Charles Y LinDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
Karl-Dimiter BissigDepartment of Pediatrics, Division of Medical Genetics, Duke University, Durham, NC 27710, USA.
Gang BaoDepartment of Bioengineering, Rice University, Houston, TX 77030, USA.
William R LagorDepartment of Molecular Physiology and Biophysics, Baylor College of Medicine, Houston, TX 77030, USA.
Baylor College of Medicine · USDuke Medical Center · USRice University · USVanderbilt University Medical Center · US

Funding

Tumor BiologyP30CA125123 · NCI · BAYLOR COLLEGE OF MEDICINE · PI Michael M Ittmann · 2007 to 2026
$73.9M
Tissue Analysis & Molecular Imaging CoreP30DK056338 · NIDDK · BAYLOR COLLEGE OF MEDICINE · PI James Versalovic · 2001 to 2026
$28.3M
Genetic Repair of Familial HypercholesterolemiaR01HL132840 · NHLBI · BAYLOR COLLEGE OF MEDICINE · PI LAGOR, WILLIAM RAYMOND · 2017 to 2025
$5.4M
BCM-Rice resource for the analysis of somatic gene editing in miceU42OD026645 · OD · BAYLOR COLLEGE OF MEDICINE · PI DICKINSON, MARY E, HEANEY, JASON D. · 2018 to 2022
$3.5M
Therapeutic potential of CRISPR/Cas9 genome engineering in humanized mouse modelsR01HL134510 · NHLBI · DUKE UNIVERSITY · PI BISSIG, KARL-DIMITER · 2016 to 2020
$3.0M
Velcro AAV Vector for tissue-specific delivery of genome editing reagents with enhanced cargo capacityUG3HL151545 · NHLBI · RICE UNIVERSITY · PI BAO, GANG, LAGOR, WILLIAM RAYMOND · 2019 to 2021
$2.3M
Therapeutic pathway reprogramming for metabolic liver diseaseR01DK115461 · NIDDK · DUKE UNIVERSITY · PI BISSIG, KARL-DIMITER · 2018 to 2020
$1.1M
Therapeutic pathway reprogramming for metabolic liver diseaseR56DK115461 · NIDDK · BAYLOR COLLEGE OF MEDICINE · PI BISSIG, KARL-DIMITER · 2017 to 2017
$238k
American Heart Association-American Stroke Association 19POST34430092NCI NIH HHS P30 CA125123NHLBI NIH HHS R01 HL132840NHLBI NIH HHS R01 HL134510NHLBI NIH HHS UG3 HL151545NIDDK NIH HHS P30 DK056338NIDDK NIH HHS R01 DK115461NIDDK NIH HHS R56 DK115461NIH HHS U42 OD026645
6 · The paper itself

Abstract

Clinical application of somatic genome editing requires therapeutics that are generalizable to a broad range of patients. Targeted insertion of promoterless transgenes can ensure that edits are permanent and broadly applicable while minimizing risks of off-target integration. In the liver, the Albumin (

Indexed as

AAVApoa1CRISPR-Cas9gene targetinggene therapygenome editinginherited metabolic disordersliver

Identifiers

PMID34141821
PMCPMC8166646
OpenAlexW3159580841

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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 OpenQuestion graph.