Evidence map›Paper›PMID 28895846›Full record

ReviewHematology/oncology clinics of North America2017

In Vivo Hematopoietic Stem Cell Transduction.

Maximilian Richter, Daniel Stone, Carol Miao, Olivier Humbert, Hans-Peter Kiem, Thalia Papayannopoulou, André Lieber

Open access · greenAbstract readReview
In one paragraph

Review in Hematology/oncology clinics of North America, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.

0numbers the graph read from it
0cells of the map it votes in
19citing papers in PubMed
3.1field-weighted citation impact, top 8% 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

19 citing papers in PubMed, 30 citations in OpenAlex.

  1. Review
  2. Article
  3. Review
  4. Review
  5. Review
  6. In vivo somatic cell base editing and prime editing.Molecular therapy : the journal of the American Society of Gene Therapy · 2021
    Review
  7. Review
  8. Maternal microchimerism and cell-mediated immune-modulation enhance engraftment following semi-allogenic intrauterine transplantation.FASEB journal : official publication of the Federation of American Societies for Experimental Biology · 2021
    Article
  9. Article
  10. Article
  11. Review
  12. Adenovirus-Antibody Complexes Contributed to Lethal Systemic Inflammation in a Gene Therapy Trial.Molecular therapy : the journal of the American Society of Gene Therapy · 2020
    Article
  13. Article
  14. Review
  15. Review
  16. Article
  17. Article
  18. HDAd5/35Molecular therapy. Methods & clinical development · 2018
    Article
  19. 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

7 authors at 4 institutions in 1 country.

Maximilian RichterDivision of Medical Genetics, University of Washington, 1705 NE Pacific Street, Seattle, WA 98195, USA.
Daniel StoneVaccine and Infectious Disease Division, Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue N, Seattle, WA 98109, USA.
Carol MiaoDepartment of Pediatrics, University of Washington, 1705 NE Pacific Street, Seattle, WA 98195, USA; Center for Immunity and Immunotherapy, Research Institute, Seattle Children's Hospital, 1900 9th Avenue, Seattle, WA 98101, USA.
Olivier HumbertClinical Research Division, Fred Hutchinson Cancer Research Center, 1100 Fairview Aveune N, Seattle, WA 98109, USA.
Hans-Peter KiemClinical Research Division, Fred Hutchinson Cancer Research Center, 1100 Fairview Aveune N, Seattle, WA 98109, USA; Department of Medicine, University of Washington School of Medicine, 1705 NE Pacific Street, Seattle, WA 98195, USA.
Thalia PapayannopoulouDivision of Hematology, University of Washington, 1705 NE Pacific Street, Seattle, WA 98195, USA.
André LieberDivision of Medical Genetics, University of Washington, 1705 NE Pacific Street, Seattle, WA 98195, USA; Department of Pathology, University of Washington, 1705 NE Pacific Street, Seattle, WA 98195, USA. Electronic address: lieber00@uw.edu.
University of Washington · USFred Hutch Cancer Center · USSeattle Children's Hospital · USUniversity of Washington Medical Center · US

Funding

In Vivo Hematopoietic Stem Cell Gene Therapy of Beta-Thalassemia and Sickle Cell DiseaseR01HL130040 · NHLBI · UNIVERSITY OF WASHINGTON · PI LIEBER, ANDRE MICHAEL · 2016 to 2024
$4.3M
Direct in vivo bone marrow transfer of lentiviral vector to correct hemophilia AR01HL123326 · NHLBI · SEATTLE CHILDREN'S HOSPITAL · PI MIAO, CAROL H · 2016 to 2019
$1.9M
GENOME EDITING FOR DEVELOPING A TREATMENT FOR BETA GLOBIN DISORDERSR01DK101328 · NIDDK · UNIVERSITY OF WASHINGTON · PI STAMATOYANNOPOULOS, THALIA · 2014 to 2018
$1.7M
Sten Cell Gene Therapy of Breast CancerR01CA136487 · NCI · UNIVERSITY OF WASHINGTON · PI LIEBER, ANDRE MICHAEL · 2009 to 2013
$1.6M
NCI NIH HHS R01 CA136487NHLBI NIH HHS R01 HL123326NHLBI NIH HHS R01 HL130040NIDDK NIH HHS R01 DK101328
6 · The paper itself

Abstract

Current protocols for hematopoietic stem cell (HSC) gene therapy, involving the transplantation of ex vivo lentivirus vector-transduced HSCs into myeloablated recipients, are complex and not without risk for the patient. In vivo HSC gene therapy can be achieved by the direct modification of HSCs in the bone marrow after intraosseous injection of gene delivery vectors. A recently developed approach involves the mobilization of HSCs from the bone marrow into peripheral the blood circulation, intravenous vector injection, and re-engraftment of genetically modified HSCs in the bone marrow. We provide examples for in vivo HSC gene therapy and discuss advantages and disadvantages.

Indexed as

Genetic TherapyTransduction, GeneticAnimalsBone Marrow CellsGene EditingGene ExpressionGenetic VectorsGene Transfer TechniquesHematopoietic Stem Cell MobilizationHematopoietic Stem CellsHumansTransgenesIntraossealIntravenousMobilizationViral vectors

Identifiers

PMID28895846
PMCPMC5659626
OpenAlexW2753217443

What OpenQuestion holds

Textmetadata
LicenceTDM
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.