Evidence map›Paper›PMID 38946936›Full record

ArticleMolecular therapy. Methods & clinical development2024

Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy.

Stuart Ellison, Karen Buckland, Yuko Learmonth, Victoria Day, Spandan Kalra, Lauren Howe, Francisco José Roman-Rodriguez, Jose Bonafont, Laura Booth, Rebecca Holley and 5 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Article
  2. Article
  3. Lentiviral Vector-MediatedHuman gene therapy · 2024
    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

15 authors.

Stuart EllisonStem Cell & Neurotherapies Group, University of Manchester, Manchester, UK.
Karen BucklandUCL Great Ormond Street Institute of Child Health, London, UK.
Yuko LearmonthStem Cell & Neurotherapies Group, University of Manchester, Manchester, UK.
Victoria DayCellular and Molecular Therapies, NHSBT Barnsley, Barnsley, UK.
Spandan KalraCellular and Molecular Therapies, NHSBT Barnsley, Barnsley, UK.
Lauren HoweCellular and Molecular Therapies, NHSBT Barnsley, Barnsley, UK.
Francisco José Roman-RodriguezUCL Great Ormond Street Institute of Child Health, London, UK.
Jose BonafontUCL Great Ormond Street Institute of Child Health, London, UK.
Laura BoothStem Cell & Neurotherapies Group, University of Manchester, Manchester, UK.
Rebecca HolleyStem Cell & Neurotherapies Group, University of Manchester, Manchester, UK.
Jon SmytheCellular and Molecular Therapies, NHSBT Barnsley, Barnsley, UK.
Simon JonesManchester University NHS Foundation Trust, Manchester, UK.
Adrian ThrasherUCL Great Ormond Street Institute of Child Health, London, UK.
Claire BoothUCL Great Ormond Street Institute of Child Health, London, UK.
Brian W BiggerStem Cell & Neurotherapies Group, University of Manchester, Manchester, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Hematopoietic stem cell gene therapy (HSCGT) is a promising therapeutic strategy for the treatment of neurodegenerative, metabolic disorders. The approach involves the

Indexed as

cleanroom validation studiesGMP cell manufacturinghCD34+ cell transductionhematopoietic stem cell gene therapyinvestigational medicinal productlentiviral vectorMPSIImucopolysaccharidosistransduction enhancers

Identifiers

PMID38946936
PMCPMC11214401

What OpenQuestion holds

Textmetadata
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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.