Evidence map›Paper›PMID 40932993›Full record

ArticleMolecular therapy. Methods & clinical development2025

GroβT rapidly and reliably mobilizes primitive hematopoietic stem and progenitor cells in nonhuman primates.

Andrea Repele, Dnyanada Pande, Mark R Enstrom, Anai Michelle Perez, Margaret Cui, Ravishankar Madhu, Veronica Nelson, Hans-Peter Kiem, Stefan Radtke

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.

Andrea RepeleTranslational Science and Therapeutics, Fred Hutchinson Cancer Center, Seattle, WA 98109, USA.
Dnyanada PandeTranslational Science and Therapeutics, Fred Hutchinson Cancer Center, Seattle, WA 98109, USA.
Mark R EnstromTranslational Science and Therapeutics, Fred Hutchinson Cancer Center, Seattle, WA 98109, USA.
Anai Michelle PerezTranslational Science and Therapeutics, Fred Hutchinson Cancer Center, Seattle, WA 98109, USA.
Margaret CuiTranslational Science and Therapeutics, Fred Hutchinson Cancer Center, Seattle, WA 98109, USA.
Ravishankar MadhuTranslational Science and Therapeutics, Fred Hutchinson Cancer Center, Seattle, WA 98109, USA.
Veronica NelsonTranslational Science and Therapeutics, Fred Hutchinson Cancer Center, Seattle, WA 98109, USA.
Hans-Peter KiemTranslational Science and Therapeutics, Fred Hutchinson Cancer Center, Seattle, WA 98109, USA.
Stefan RadtkeTranslational Science and Therapeutics, Fred Hutchinson Cancer Center, Seattle, WA 98109, USA.

Funding

Upgrade of confocal microscopy at the Oregon National Primate Research CenterP51OD011092 · OD · OREGON HEALTH & SCIENCE UNIVERSITY · PI Bonnie J. Nagel · 2012 to 2026
$203.9M
Washington National Primate Research CenterP51OD010425 · OD · UNIVERSITY OF WASHINGTON · PI Mari Ostendorf · 2012 to 2026
$201.8M
Novel Gene Editing Approaches for HemoglobinopathiesR01HL136135 · NHLBI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI HANS-PETER KIEM · 2017 to 2026
$7.7M
Targeting a Novel Hematopoietic Stem Cell Population in Non-Human Primates for Effective and Sustained Gene TherapyR01AI135953 · NIAID · FRED HUTCHINSON CANCER RESEARCH CENTER · PI KIEM, HANS-PETER · 2018 to 2022
$4.5M
NHLBI NIH HHS R01 HL136135NIAID NIH HHS R01 AI135953NIH HHS P51 OD010425NIH HHS P51 OD011092
6 · The paper itself

Abstract

Autologous hematopoietic stem cell (HSC) gene therapy has gone through remarkable advancements in recent years, especially for the treatment of sickle cell disease (SCD). However, the collection of HSCs from SCD patients requires unique considerations, as granulocyte colony-stimulating factor (G-CSF)-mediated mobilization is contraindicated, and plerixafor-only mobilization is highly variable. Consequently, alternative mobilization regimens that are safe for SCD patients and generate better cell yields are desirable for SCD HSC gene therapy. Here, we evaluated a combination of plerixafor (AMD3100, a CXCR4 antagonist) with GroβT (MGTA-145/GroβT, a CXCR2 agonist) against the current gold-standard G-CSF for HSC gene therapy in nonhuman primates (NHPs) for HSC mobilization, leukapheresis,

Indexed as

AMD3100bone marrow nicheex vivo gene therapyfetal hemoglobin reactivationG-CSFGroβTHSCleukapheresisNHPsickle cell disease

Identifiers

PMID40932993
PMCPMC12418998

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.