Evidence map›Paper›PMID 29100011›Full record

ReviewCell stem cell2017

Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned.

Richard A Morgan, David Gray, Anastasia Lomova, Donald B Kohn

Abstract readReview
In one paragraph

Review in Cell stem cell, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 145 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
145citing papers in PubMed, 1 pooled it
–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

145 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Electroporation for High-Efficiency Delivery of CRISPR to Hematopoietic Cells.Methods in molecular biology (Clifton, N.J.) · 2027
    Article
  3. Review
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  18. Quantifying the mutational landscape of retroviral and lentiviral vectors in gene therapy patients.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  19. Article
  20. Article

85 more citing papers are in PubMed but not listed here.

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

4 authors.

Richard A MorganCharles R. Drew University of Medicine and Science, Los Angeles, CA, 90059; Department of Molecular and Medical Pharmacology, David Geffen School of Medicine at University of California, Los Angeles, CA, 90095.
David GrayMolecular Biology Institute Interdepartmental Doctoral Program, University of California, Los Angeles, CA, 90095.
Anastasia LomovaDepartment of Molecular and Medical Pharmacology, David Geffen School of Medicine at University of California, Los Angeles, CA, 90095.
Donald B KohnDepartment of Molecular and Medical Pharmacology, David Geffen School of Medicine at University of California, Los Angeles, CA, 90095; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, CA, 90095; Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles, CA, 90095; The Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, CA, USA. Electronic address: dkohn1@mednet.ucla.edu.

Funding

Interdisciplinary Training in Virology and Gene TherapyT32AI060567 · NIAID · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI WU, TING-TING · 2004 to 2018
$3.4M
Improved Globin Expression Vectors for Gene Therapy of Human HemoglobinopathiesF31HL134313 · NHLBI · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI MORGAN, RICHARD A · 2016 to 2020
$211k
Improving targeted nuclease-mediated gene correction in human hematopoietic stem cellsF31HL134208 · NHLBI · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI LOMOVA, ANASTASIA · 2017 to 2018
$75k
NHLBI NIH HHS F31 HL134208NHLBI NIH HHS F31 HL134313NIAID NIH HHS T32 AI060567
6 · The paper itself

Abstract

The use of allogeneic hematopoietic stem cells (HSCs) to treat genetic blood cell diseases has become a clinical standard but is limited by the availability of suitable matched donors and potential immunologic complications. Gene therapy using autologous HSCs should avoid these limitations and thus may be safer. Progressive improvements in techniques for genetic correction of HSCs, by either vector gene addition or gene editing, are facilitating successful treatments for an increasing number of diseases. We highlight the progress, successes, and remaining challenges toward the development of HSC gene therapies and discuss lessons they provide for the development of future clinical stem cell therapies.

Indexed as

Genetic TherapyClinical Trials as TopicGene EditingGenetic VectorsGene Transfer TechniquesHematopoietic Stem CellsHematopoietic Stem Cell TransplantationHumans

Identifiers

PMID29100011
PMCPMC6039108

What OpenQuestion holds

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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.