Evidence map›Paper›PMID 38108962›Full record

ArticleMethods in molecular biology (Clifton, N.J.)2024

Alpha-1 Antitrypsin Deficiency.

Alisha M Gruntman, Wen Xue, Terence R Flotte

Abstract read
PubMed Publisher
In one paragraph

Article in Methods in molecular biology (Clifton, N.J.), 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
4.6field-weighted citation impact, top 4% 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

2 citing papers in PubMed, 7 citations in OpenAlex.

  1. Review
  2. 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

3 authors at 1 institution in 1 country.

Alisha M GruntmanDepartment of Pediatrics, University of Massachusetts Chan Medical School, Worcester, MA, USA.
Wen XueDepartment of Pediatrics, University of Massachusetts Chan Medical School, Worcester, MA, USA.
Terence R FlotteDepartment of Pediatrics, University of Massachusetts Chan Medical School, Worcester, MA, USA. terry.flotte@umassmed.edu.
University of Massachusetts Chan Medical School · US

Funding

Vector Immunology CoreP01HL158506 · NHLBI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Dan Wang · 2021 to 2026
$19.5M
NHLBI NIH HHS P01 HL158506
6 · The paper itself

Abstract

Alpha-1 antitrypsin (AAT) deficiency is a common monogenic disorder in which there is a strong founder effect of a single missense mutation in SERPINA1, the gene encoding this major circulating serum anti-protease that is normally expressed primarily in hepatocytes. These features make AAT deficiency particularly attractive as a target for therapeutic gene editing using a wide variety of approaches.

Indexed as

alpha 1-Antitrypsin DeficiencyEndopeptidasesFounder EffectGene EditingHepatocytesHumansEndopeptidasesAlpha-1 antitrypsinCRISPR–Cas9EmphysemaGene editingGene therapyLiverLungVector

Identifiers

PMID38108962
OpenAlexW4389895470

What OpenQuestion holds

Textmetadata
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.