Evidence map›Paper›PMID 35795780›Full record

ArticleMolecular therapy. Methods & clinical development2022

Mutant allele knockout with novel CRISPR nuclease promotes myelopoiesis in ELANE neutropenia.

Peter Sabo, Vahagn Makaryan, Yosef Dicken, Lital Povodovski, Liat Rockah, Tzlil Bar, Matan Gabay, Dalia Elinger, Ella Segal, Ora Haimov and 6 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
1.9field-weighted citation impact, top 14% 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

9 citing papers in PubMed, 11 citations in OpenAlex.

  1. Review
  2. Targeted inhibition ofMolecular therapy. Methods & clinical development · 2025
    Article
  3. ModelingJournal of cellular immunology · 2025
    Article
  4. Article
  5. CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Article
  6. Review
  7. Article
  8. Review
  9. Review
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

16 authors at 2 institutions in 1 country.

Peter SaboDepartment of Medicine, University of Washington, Box 356422, 1959 NE Pacific Street, Room AA522, Seattle, WA 98195, USA.
Vahagn MakaryanDepartment of Medicine, University of Washington, Box 356422, 1959 NE Pacific Street, Room AA522, Seattle, WA 98195, USA.
Yosef DickenEmendoBio, Inc., 400 W 61 Street, #2330, New York NY 10069, USA.
Lital PovodovskiEmendoBio, Inc., 400 W 61 Street, #2330, New York NY 10069, USA.
Liat RockahEmendoBio, Inc., 400 W 61 Street, #2330, New York NY 10069, USA.
Tzlil BarEmendoBio, Inc., 400 W 61 Street, #2330, New York NY 10069, USA.
Matan GabayEmendoBio, Inc., 400 W 61 Street, #2330, New York NY 10069, USA.
Dalia ElingerEmendoBio, Inc., 400 W 61 Street, #2330, New York NY 10069, USA.
Ella SegalEmendoBio, Inc., 400 W 61 Street, #2330, New York NY 10069, USA.
Ora HaimovEmendoBio, Inc., 400 W 61 Street, #2330, New York NY 10069, USA.
Maya AntoshviliEmendoBio, Inc., 400 W 61 Street, #2330, New York NY 10069, USA.
Anat London DroriEmendoBio, Inc., 400 W 61 Street, #2330, New York NY 10069, USA.
Tanoya PoulsenDepartment of Medicine, University of Washington, Box 356422, 1959 NE Pacific Street, Room AA522, Seattle, WA 98195, USA.
Asael HermanEmendoBio, Inc., 400 W 61 Street, #2330, New York NY 10069, USA.
Rafi EmmanuelEmendoBio, Inc., 400 W 61 Street, #2330, New York NY 10069, USA.
David C DaleDepartment of Medicine, University of Washington, Box 356422, 1959 NE Pacific Street, Room AA522, Seattle, WA 98195, USA.
EngenderHealth · USUniversity of Washington · US

Funding

Severe Chronic Neutropenia International RegistryR24AI049393 · NIAID · UNIVERSITY OF WASHINGTON · PI DALE, DAVID CHANDLER · 2004 to 2019
$10.6M
Gene Editing vs Neutrophil Elastase Inhibitors for Treatment of ELANE Associated NeutropeniaR01HL151629 · NHLBI · UNIVERSITY OF WASHINGTON · PI DALE, DAVID CHANDLER · 2020 to 2022
$1.8M
NHLBI NIH HHS R01 HL151629NIAID NIH HHS R24 AI049393
6 · The paper itself

Abstract

Severe congenital neutropenia (SCN) is a life-threatening marrow failure disorder, usually caused by heterozygous mutations in

Indexed as

allele-specific editingCRISPRELANEgene editingneutropenianeutrophils

Identifiers

PMID35795780
PMCPMC9240714
OpenAlexW4282924475

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.