Evidence map›Paper›PMID 37568039›Full record

ArticleGene therapy2023

Biodistribution of lentiviral transduced adipose-derived stem cells for "ex-vivo" regional gene therapy for bone repair.

Jennifer A Bell, Kevin Collon, Cory Mayfield, Matthew C Gallo, Stephanie W Chang, Osamu Sugiyama, Amy H Tang, Roger Hollis, Shefali Chopra, Donald B Kohn and 1 more

Abstract read
PubMed Publisher
In one paragraph

Article in Gene therapy, 2023. 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
1.9field-weighted citation impact, top 13% of its field
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, 6 citations in OpenAlex.

  1. Review
  2. Article
  3. Review
  4. Bioengineering (Basel, Switzerland) · 2025
    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

11 authors at 2 institutions in 1 country.

Jennifer A BellDepartment of Orthopaedic Surgery, Keck School of Medicine of the University of Southern California, 2011 Zonal Ave, HMR 702, Los Angeles, CA, 90089, USA. Jennifer.bell@med.usc.edu.ORCID 0000-0003-0959-1401
Kevin CollonDepartment of Orthopaedic Surgery, Keck School of Medicine of the University of Southern California, 2011 Zonal Ave, HMR 702, Los Angeles, CA, 90089, USA.ORCID 0000-0001-8984-2447
Cory MayfieldDepartment of Orthopaedic Surgery, Keck School of Medicine of the University of Southern California, 2011 Zonal Ave, HMR 702, Los Angeles, CA, 90089, USA.
Matthew C GalloDepartment of Orthopaedic Surgery, Keck School of Medicine of the University of Southern California, 2011 Zonal Ave, HMR 702, Los Angeles, CA, 90089, USA.
Stephanie W ChangDepartment of Orthopaedic Surgery, Keck School of Medicine of the University of Southern California, 2011 Zonal Ave, HMR 702, Los Angeles, CA, 90089, USA.
Osamu SugiyamaDepartment of Orthopaedic Surgery, Keck School of Medicine of the University of Southern California, 2011 Zonal Ave, HMR 702, Los Angeles, CA, 90089, USA.
Amy H TangDepartment of Orthopaedic Surgery, Keck School of Medicine of the University of Southern California, 2011 Zonal Ave, HMR 702, Los Angeles, CA, 90089, USA.
Roger HollisDepartment of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, CA, USA.
Shefali ChopraDepartment of Pathology, Keck School of Medicine of University of Southern California, 1975 Zonal Ave, Los Angeles, CA, 90089, USA.
Donald B KohnDepartment of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, CA, USA.ORCID 0000-0003-1840-6087
Jay R LiebermanDepartment of Orthopaedic Surgery, Keck School of Medicine of the University of Southern California, 2011 Zonal Ave, HMR 702, Los Angeles, CA, 90089, USA.
University of Southern California · USUniversity of California, Los Angeles · US

Funding

Foundation for the National Institutes of Health (Foundation for the National Institutes of Health, Inc.) R01AR057076]
6 · The paper itself

Abstract

Ex-vivo gene therapy has been shown to be an effective method for treating bone defects in pre-clinical models. As gene therapy is explored as a potential treatment option in humans, an assessment of the safety profile becomes an important next step. The purpose of this study was to evaluate the biodistribution of viral particles at the defect site and various internal organs in a rat femoral defect model after implantation of human ASCs transduced with lentivirus (LV) with two-step transcriptional activation (TSTA) of bone morphogenetic protein-2 (LV-TSTA-BMP-2). Animals were sacrificed at 4-, 14-, 56-, and 84-days post implantation. The defects were treated with either a standard dose (SD) of 5 million cells or a high dose (HD) of 15 million cells to simulate a supratherapeutic dose. Treatment groups included (1) SD LV-TSTA-BMP-2 (2) HD LV-TSTA-BMP-2, (3) SD LV-TSTA-GFP (4) HD LV-TSTA-GFP and (5) SD nontransduced cells. The viral load at the defect site and ten organs was assessed at each timepoint. Histology of all organs, ipsilateral tibia, and femur were evaluated at each timepoint. There were nearly undetectable levels of LV-TSTA-BMP-2 transduced cells at the defect site at 84-days and no pathologic changes in any organ at all timepoints. In conclusion, human ASCs transduced with a lentiviral vector were both safe and effective in treating critical size bone defects in a pre-clinical model. These results suggest that regional gene therapy using lentiviral vector to treat bone defects has the potential to be a safe and effective treatment in humans.

Indexed as

Bone Morphogenetic Protein 2LentivirusAnimalsGenetic TherapyHumansRatsStem CellsTissue DistributionBone Morphogenetic Protein 2

Identifiers

PMID37568039
OpenAlexW4385761962

What OpenQuestion holds

Textmetadata
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.