Evidence map›Paper›PMID 35159366›Full record

ReviewCells2022

Hematopoietic Cells from Pluripotent Stem Cells: Hope and Promise for the Treatment of Inherited Blood Disorders.

Ilaria Rao, Laura Crisafulli, Marianna Paulis, Francesca Ficara

Open access · goldAbstract readReview
In one paragraph

Review in Cells, 2022. 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
1.9field-weighted citation impact, top 14% 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, 23 citations in OpenAlex.

  1. Blood Pharming of Red Cells.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2026
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  12. An Overview of Autophagy in Hematopoietic Stem Cell Transplantation.Frontiers in bioengineering and biotechnology · 2022
    Review
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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

4 authors at 2 institutions in 1 country.

Ilaria RaoIRCCS Humanitas Research Hospital, Via Manzoni 56, 20089 Rozzano, Italy.ORCID 0000-0003-2532-2957
Laura CrisafulliIRCCS Humanitas Research Hospital, Via Manzoni 56, 20089 Rozzano, Italy.
Marianna PaulisIRCCS Humanitas Research Hospital, Via Manzoni 56, 20089 Rozzano, Italy.ORCID 0000-0001-7803-9982
Francesca FicaraIRCCS Humanitas Research Hospital, Via Manzoni 56, 20089 Rozzano, Italy.ORCID 0000-0003-1873-1189
Institute of Genetic and Biomedical Research · ITHumanitas University · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Inherited blood disorders comprise a large spectrum of diseases due to germline mutations in genes with key function in the hematopoietic system; they include immunodeficiencies, anemia or metabolic diseases. For most of them the only curative treatment is bone marrow transplantation, a procedure associated to severe complications; other therapies include red blood cell and platelet transfusions, which are dependent on donor availability. An alternative option is gene therapy, in which the wild-type form of the mutated gene is delivered into autologous hematopoietic stem cells using viral vectors. A more recent therapeutic perspective is gene correction through CRISPR/Cas9-mediated gene editing, that overcomes safety concerns due to insertional mutagenesis and allows correction of base substitutions in large size genes difficult to incorporate into vectors. However, applying this technique to genomic disorders caused by large gene deletions is challenging. Chromosomal transplantation has been proposed as a solution, using a universal source of wild-type chromosomes as donor, and induced pluripotent stem cells (iPSCs) as acceptor. One of the obstacles to be addressed for translating PSC research into clinical practice is the still unsatisfactory differentiation into transplantable hematopoietic stem or mature cells. We provide an overview of the recent progresses in this field and discuss challenges and potential of iPSC-based therapies for the treatment of inherited blood disorders.

Indexed as

Blood Coagulation Disorders, InheritedCell DifferentiationGene EditingGenetic TherapyHematopoietic Stem CellsHumansPluripotent Stem Cellschromosome transplantationdifferentiationgene editinggene therapygenomic disordershematopoiesishematopoietic stem cellinherited blood disordersiPSCpluripotent stem cell

Identifiers

PMID35159366
PMCPMC8834203
OpenAlexW4210577691

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.