Evidence map›Paper›PMID 33427035›Full record

ArticleHuman gene therapy2021

Safe and Effective

Paula Cannon, Aravind Asokan, Agnieszka Czechowicz, Paula Hammond, Donald B Kohn, Andre Lieber, Punam Malik, Peter Marks, Matthew Porteus, Els Verhoeyen and 3 more

Open access · bronzeAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Human gene therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.

0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed
1.5field-weighted citation impact, top 17% 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

13 citing papers in PubMed, 20 citations in OpenAlex.

  1. Article
  2. Restoring hematopoietic stem and progenitor cell function inMolecular therapy. Nucleic acids · 2025
    Article
  3. Review
  4. Review
  5. Review
  6. Review
  7. Review
  8. Review
  9. Review
  10. Review
  11. Genome editing in large animal models.Molecular therapy : the journal of the American Society of Gene Therapy · 2021
    Review
  12. Understanding and overcoming adverse consequences of genome editing on hematopoietic stem and progenitor cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2021
    Review
  13. 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

13 authors at 11 institutions in 2 countries.

Paula CannonKeck School of Medicine, University of Southern California, Los Angeles, California, USA.
Aravind AsokanDepartment of Surgery, Duke University School of Medicine, Durham, North Carolina, USA.
Agnieszka CzechowiczDepartment of Pediatrics.
Paula HammondDepartment of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA.
Donald B KohnDepartment of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA.
Andre LieberDepartment of Medicine, University of Washington, Seattle, Washington, USA.
Punam MalikDivision of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio, USA.
Peter MarksU.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Matthew PorteusDepartment of Pediatrics.
Els VerhoeyenCIRI, Université de Lyon, INSERM, CNRS, ENS de Lyon, Lyon, France.
Drew WeissmanDepartment of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Irving WeissmanStanford Institute for Stem Cell Biology and Regenerative Medicine; Stanford University School of Medicine, Stanford, California, USA.
Hans-Peter KiemStem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, Washington, USA.
Cincinnati Children's Hospital Medical Center · USDuke University · USFred Hutch Cancer Center · USMassachusetts Institute of Technology · USStanford University · USUnited States Food and Drug Administration · USUniversité Claude Bernard Lyon 1 · FRUniversity of California, Los Angeles · USUniversity of Pennsylvania · USUniversity of Southern California · USUniversity of Washington · 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
Approach for in vivo gene delivery into hematopoietic stem cells for hemophilia A therapyR01HL141781 · NHLBI · UNIVERSITY OF WASHINGTON · PI LIEBER, ANDRE MICHAEL · 2018 to 2021
$2.3M
NHLBI NIH HHS R01 HL130040NHLBI NIH HHS R01 HL141781
6 · The paper itself

Abstract

On May 11, 2020, the National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation (Gates Foundation) held an exploratory expert scientific roundtable to inform an NIH-Gates Foundation collaboration on the development of scalable, sustainable, and accessible HIV and sickle cell disease (SCD) therapies based on

Indexed as

Anemia, Sickle CellGene EditingCell DifferentiationGenetic TherapyHematopoietic Stem CellsHumansgene editingin vivosickle cell diseasetargeting

Identifiers

PMID33427035
PMCPMC8917879
OpenAlexW3119631190

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.