Evidence map›Paper›PMID 38745893›Full record

ArticleMolecular therapy. Methods & clinical development2024

A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease.

Kevyn L Hart, Boya Liu, Devin Brown, Beatriz Campo-Fernandez, Kevin Tam, Katherine Orr, Roger P Hollis, Christian Brendel, David A Williams, Donald B Kohn

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 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
  4. Review
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

10 authors.

Kevyn L HartDepartment of Human Genetics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA 90095, USA.
Boya LiuDivision of Hematology/Oncology, Boston Children's Hospital, Boston, MA 02115, USA.
Devin BrownDepartment of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.
Beatriz Campo-FernandezDepartment of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.
Kevin TamDepartment of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.
Katherine OrrCSUN-UCLA Stem Cell Scientist Training Program, California State University, Northridge, Northridge, CA 91330, USA.
Roger P HollisDepartment of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.
Christian BrendelDivision of Hematology/Oncology, Boston Children's Hospital, Boston, MA 02115, USA.
David A WilliamsDivision of Hematology/Oncology, Boston Children's Hospital, Boston, MA 02115, USA.
Donald B KohnDepartment of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.

Funding

Training CoreTL1DK132768 · NIDDK · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI Elizabeta Nemeth · 2021 to 2026
$3.4M
NIDDK NIH HHS TL1 DK132768
6 · The paper itself

Abstract

A major limitation of gene therapy for sickle cell disease (SCD) is the availability and access to a potentially curative one-time treatment, due to high treatment costs. We have developed a high-titer bifunctional lentiviral vector (LVV) in a vector backbone that has reduced size, high vector yields, and efficient gene transfer to human CD34

Indexed as

anti-sickling hemoglobinBCL11Agene therapyhematopoietic stem cellslentiviral vectorshmiRsickle cell disease

Identifiers

PMID38745893
PMCPMC11091523

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Registered trials

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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.