Evidence map›Paper›PMID 18063756›Full record

ArticleScience (New York, N.Y.)2007

Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin.

Jacob Hanna, Marius Wernig, Styliani Markoulaki, Chiao-Wang Sun, Alexander Meissner, John P Cassady, Caroline Beard, Tobias Brambrink, Li-Chen Wu, Tim M Townes and 1 more

Registry-linked trialAbstract read
PubMed Publisher
In one paragraph

Article in Science (New York, N.Y.), 2007. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00840567 (Acquisition of Skin Biopsies and Blood Samples From Normal Volunteers and Patients With Benign, Inherited Hematologic Diseases for Research Purposes), which is not on this map. Cited by 580 papers.

0numbers the graph read from it
0cells of the map it votes in
580citing papers in PubMed
70.4field-weighted citation impact, top 1% 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.

NCT00840567 naterminatednot on this mapstarted 2009, after this paper: background citation

Acquisition of Skin Biopsies and Blood Samples From Normal Volunteers and Patients With Benign, Inherited Hematologic Diseases for Research Purposes

TypeinterventionalSponsorWashington University School of MedicineRan2009 to 2010Enrolled7ConditionsAnemia, Sickle CellArmsSkin biopsy, Blood draw & sample
3 · Its place in the literature

Who cites it

580 citing papers in PubMed, 1,490 citations in OpenAlex.

  1. Review
  2. Article
  3. Article
  4. Article
  5. Article
  6. Article
  7. Treating genetic blood disorders in the era of CRISPR-mediated genome editing.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  8. Review
  9. Article
  10. Article
  11. Article
  12. Review
  13. Thymidylate synthase disruption to limit cell proliferation in cell therapies.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Article
  14. Review
  15. Review
  16. Article
  17. Review
  18. Review
  19. Article
  20. Gene Editing and Human iPSCs in Cardiovascular and Metabolic Diseases.Advances in experimental medicine and biology · 2023
    Article

520 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

11 authors at 3 institutions in 1 country.

Jacob HannaWhitehead Institute for Biomedical Research, Cambridge, MA 02142, USA.
Marius Wernig
Styliani Markoulaki
Chiao-Wang Sun
Alexander Meissner
John P Cassady
Caroline Beard
Tobias Brambrink
Li-Chen Wu
Tim M Townes
Rudolf Jaenisch
Whitehead Institute for Biomedical Research · USMassachusetts Institute of Technology · USUniversity of Alabama at Birmingham · US

Funding

GENOMIC IMPRINTING AND THE CLONING OF MICER37CA084198 · NCI · WHITEHEAD INSTITUTE FOR BIOMEDICAL RES · PI JAENISCH, RUDOLF · 2000 to 2008
$9.2M
Epigenetics, Stem Cells, and CancerR01CA087869 · NCI · WHITEHEAD INSTITUTE FOR BIOMEDICAL RES · PI JAENISCH, RUDOLF · 2001 to 2010
$8.2M
Mouse Model of Sickle Cell Disease and Genetic TherapiesR01HL057619 · NHLBI · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI TOWNES, TIM M. · 1996 to 2014
$5.3M
NCI NIH HHS 5-R37-CA084198NCI NIH HHS 5-RO1-CA087869NHLBI NIH HHS 2-R01-HL057619PHS HHS 5-RO1-HDO45022
6 · The paper itself

Abstract

It has recently been demonstrated that mouse and human fibroblasts can be reprogrammed into an embryonic stem cell-like state by introducing combinations of four transcription factors. However, the therapeutic potential of such induced pluripotent stem (iPS) cells remained undefined. By using a humanized sickle cell anemia mouse model, we show that mice can be rescued after transplantation with hematopoietic progenitors obtained in vitro from autologous iPS cells. This was achieved after correction of the human sickle hemoglobin allele by gene-specific targeting. Our results provide proof of principle for using transcription factor-induced reprogramming combined with gene and cell therapy for disease treatment in mice. The problems associated with using retroviruses and oncogenes for reprogramming need to be resolved before iPS cells can be considered for human therapy.

Indexed as

Cellular ReprogrammingHematopoietic Stem Cell TransplantationAnemia, Sickle CellAnimalsCell DifferentiationCells, CulturedDisease Models, AnimalDNA-Binding ProteinsEmbryonic Stem CellsErythrocyte CountFibroblastsGenes, mycGlobinsHematopoiesisHematopoietic Stem CellsHemoglobin ADNA-Binding ProteinsGlobinsHemoglobin AHemoglobin, SickleKruppel-Like Factor 4Kruppel-Like Transcription FactorsOctamer Transcription Factor-3Pou5f1 protein, mouseSOX2 protein, humanSox2 protein, mouseSOXB1 Transcription FactorsTrans-Activators

Identifiers

PMID18063756
OpenAlexW2104978512

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

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.