Evidence map›Paper›PMID 38108963›Full record

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

Approaches to Therapeutic Gene Editing in 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 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

3 authors.

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.

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

Five distinct gene therapy approaches have been developed for treating AATD. These approaches include knockout of the mutant (PiZ) allele by introduction of double-strand breaks (DSBs) and subsequent creation of insertions and deletions (indels) by DSB repair, homology-directed repair (HDR) targeted to the mutation site, base editing, prime editing, and alternatively targeted knock-in techniques. Each approach will be discussed and a brief summary of a standard CRISPR-Cas9 targeting method will be presented.

Indexed as

alpha 1-Antitrypsin DeficiencyGene EditingAllelesGenetic TherapyHumansINDEL MutationMutationBase editingDouble-strand breaksHomology-directed repairIndelsPrime editing

Identifiers

PMID38108963

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.