Evidence map›Paper›PMID 40176947›Full record

ArticleMolecular therapy. Methods & clinical development2025

Preclinical efficacy of a modified gamma-globin lentivirus gene therapy in Berkeley sickle cell anemia mice and human xenograft models.

Archana Shrestha, Devin M Pillis, Sydney Felker, Mengna Chi, Kimberly Wagner, Oluwabukola T Gbotosho, Joseph Sieling, Mohammad Shadid, Punam Malik

Abstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2025. 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

9 authors.

Archana ShresthaDivision of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center (CCHMC), Cincinnati, OH 45229, USA.
Devin M PillisDivision of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center (CCHMC), Cincinnati, OH 45229, USA.
Sydney FelkerDivision of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center (CCHMC), Cincinnati, OH 45229, USA.
Mengna ChiDivision of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center (CCHMC), Cincinnati, OH 45229, USA.
Kimberly WagnerDivision of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center (CCHMC), Cincinnati, OH 45229, USA.
Oluwabukola T GbotoshoDivision of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center (CCHMC), Cincinnati, OH 45229, USA.
Joseph SielingAruvant Sciences, New York, NY 10036, USA.
Mohammad ShadidAruvant Sciences, New York, NY 10036, USA.
Punam MalikDivision of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center (CCHMC), Cincinnati, OH 45229, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

We previously showed correction of sickle cell anemia (SCA) in mice utilizing a lentiviral vector (LV) expressing human γ-globin. Herein, we made a G16D mutation in the γ-globin gene to generate the G16D mutation (GbG

Indexed as

CD34+ gene transferengraftmentfetal hemoglobinhematopoietic stem cellhuman gamma-globininsulatorlentiviral vectorpreclinical studysickle cell anemia

Identifiers

PMID40176947
PMCPMC11964741

What OpenQuestion holds

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LicenceCC BY-NC-ND
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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.