Evidence map›Paper›PMID 32226796›Full record

ArticleMolecular therapy. Methods & clinical development2020

Effective Multi-lineage Engraftment in a Mouse Model of Fanconi Anemia Using Non-genotoxic Antibody-Based Conditioning.

Meera A Srikanthan, Olivier Humbert, Kevin G Haworth, Christina Ironside, Yogendra S Rajawat, Bruce R Blazar, Rahul Palchaudhuri, Anthony E Boitano, Michael P Cooke, David T Scadden and 1 more

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
24citing papers in PubMed, 1 pooled it
3.4field-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

24 citing papers in PubMed, 1 synthesis or guideline pooled it, 30 citations in OpenAlex.

  1. Pooled it
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  11. Correction of osteopetrosis in the neonateFrontiers in endocrinology · 2024
    Article
  12. Article
  13. Efficient manufacturing and engraftment ofMolecular therapy. Methods & clinical development · 2023
    Article
  14. Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic LeukodystrophyThe Journal of neuroscience : the official journal of the Society for Neuroscience · 2023
    Article
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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 5 institutions in 1 country.

Meera A SrikanthanStem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA, USA.
Olivier HumbertStem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA, USA.
Kevin G HaworthStem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA, USA.
Christina IronsideStem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA, USA.
Yogendra S RajawatStem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA, USA.
Bruce R BlazarDepartment of Pediatrics, Division of Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN, USA.
Rahul PalchaudhuriMagenta Therapeutics, Cambridge, MA, USA.
Anthony E BoitanoMagenta Therapeutics, Cambridge, MA, USA.
Michael P CookeMagenta Therapeutics, Cambridge, MA, USA.
David T ScaddenDepartment of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA, USA.
Hans-Peter KiemStem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA, USA.
Fred Hutch Cancer Center · USFate Therapeutics (United States) · USHarvard University · USUniversity of Minnesota · USUniversity of Washington · US

Funding

Translational Bioimaging Core Shared ResourceP30CA015704 · NCI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI Eric Collisson · 1985 to 2026
$296.4M
Nongenotoxic conditioning for gene therapy and allogeneic transplantation in Fanconi anemiaR01HL147324 · NHLBI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI Bruce R Blazar, HANS-PETER KIEM · 2020 to 2026
$4.1M
NCI NIH HHS P30 CA015704NHLBI NIH HHS R01 HL147324
6 · The paper itself

Abstract

Conditioning chemotherapy is used to deplete hematopoietic stem cells in the recipient's marrow, facilitating donor cell engraftment. Although effective, a major issue with chemotherapy is the systemic genotoxicity that increases the risk for secondary malignancies. Antibody conjugates targeting hematopoietic cells are an emerging non-genotoxic method of opening the marrow niche and promoting engraftment of transplanted cells while maintaining intact marrow cellularity. Specifically, this platform would be useful in diseases associated with DNA damage or cancer predisposition, such as dyskeratosis congenita, Schwachman-Diamond syndrome, and Fanconi anemia (FA). Our approach utilizes antibody-drug conjugates (ADC) as an alternative conditioning regimen in an FA mouse model of autologous transplantation. Antibodies targeting either CD45 or CD117 were conjugated to saporin (SAP), a ribosomal toxin.

Indexed as

CD117Fanconi anemiaFanconi anemia gene therapynon-genotoxic conditioningsaporin

Identifiers

PMID32226796
PMCPMC7096734
OpenAlexW3005526803

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.