Letter to the Editor: Production of Mature Healthy Hematopoietic Cells from Induced Pluripotent Stem Cells Derived from an AML Diagnostic Sample Containing the t(8;21) Translocation

Hoffmann, Dirk; Göhring, Gudrun; Heuser, Michael; Ganser, Arnold; Schambach, Axel; Morgan, Michael

doi:10.1002/stem.2207

Cited by 6 publications

(3 citation statements)

References 8 publications

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“…Moreover, the malignant genetic or cytogenetic transformations have shown to interfere in the reprogramming of disease cells. Attempts to reprogram cells from AML patients with high-risk translocations resulted in cytogenetically normal iPSCs [77,78] suggesting that not all genetic abnormalities can be preserved during reprogramming. This can be seen in relatively negligible reports of iPSC generations from fully malignant cells as compared to pre-malignant cells as in case of DS-ML and TMD.…”

Section: Disease Modeling Using Patient-derived Ipscsmentioning

confidence: 99%

Harnessing the Power of Induced Pluripotent Stem Cells and Gene Editing Technology: Therapeutic Implications in Hematological Malignancies

Sidhu

Barwe

Pillai

et al. 2021

Cells

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In vitro modeling of hematological malignancies not only provides insights into the influence of genetic aberrations on cellular and molecular mechanisms involved in disease progression but also aids development and evaluation of therapeutic agents. Owing to their self-renewal and differentiation capacity, induced pluripotent stem cells (iPSCs) have emerged as a potential source of short in supply disease-specific human cells of the hematopoietic lineage. Patient-derived iPSCs can recapitulate the disease severity and spectrum of prognosis dictated by the genetic variation among patients and can be used for drug screening and studying clonal evolution. However, this approach lacks the ability to model the early phases of the disease leading to cancer. The advent of genetic editing technology has promoted the generation of precise isogenic iPSC disease models to address questions regarding the underlying genetic mechanism of disease initiation and progression. In this review, we discuss the use of iPSC disease modeling in hematological diseases, where there is lack of patient sample availability and/or difficulty of engraftment to generate animal models. Furthermore, we describe the power of combining iPSC and precise gene editing to elucidate the underlying mechanism of initiation and progression of various hematological malignancies. Finally, we discuss the power of iPSC disease modeling in developing and testing novel therapies in a high throughput setting.

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Section: Disease Modeling Using Patient-derived Ipscsmentioning

confidence: 99%

Harnessing the Power of Induced Pluripotent Stem Cells and Gene Editing Technology: Therapeutic Implications in Hematological Malignancies

Sidhu

Barwe

Pillai

et al. 2021

Cells

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show abstract

“…The iPSC lines derived did not harbor the translocation and were cytogenetically normal ( Yamasaki et al, 2019 ). Similarly, reprogramming an AML patient sample with a t(8;21) translocation resulted in only the successful reprogramming of cytogenetically normal iPSC lines ( Hoffmann et al, 2016 ). These studies provide further evidence that particular chromosomal abnormalities and point mutations are selected against during reprogramming.…”

Section: Barriers To Reprogrammingmentioning

confidence: 99%

Induced pluripotent stem cell models of myeloid malignancies and clonal evolution

Reilly

Doulatov

2021

Stem Cell Research

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“…AML cells appear to reprogram with even lower efficiency, presumably because of a higher burden of genetic and epigenetic abnormalities (Chao et al, 2017; Kotini et al, 2017; Hoffmann et al, 2016; Salci et al, 2015; Lee et al, 2017). Regardless, AML-iPSC lines have successfully been derived and studied.…”

Section: Ipsc Models Of Hematologic Disordersmentioning

confidence: 99%

Modeling blood diseases with human induced pluripotent stem cells

Georgomanoli

Papapetrou

2019

Disease Models &Amp; Mechanisms

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Induced pluripotent stem cells (iPSCs) are derived from somatic cells through a reprogramming process, which converts them to a pluripotent state, akin to that of embryonic stem cells. Over the past decade, iPSC models have found increasing applications in the study of human diseases, with blood disorders featuring prominently. Here, we discuss methodological aspects pertaining to iPSC generation, hematopoietic differentiation and gene editing, and provide an overview of uses of iPSCs in modeling the cell and gene therapy of inherited genetic blood disorders, as well as their more recent use as models of myeloid malignancies. We also discuss the strengths and limitations of iPSCs compared to model organisms and other cellular systems commonly used in hematology research.

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Letter to the Editor: Production of Mature Healthy Hematopoietic Cells from Induced Pluripotent Stem Cells Derived from an AML Diagnostic Sample Containing the t(8;21) Translocation

Cited by 6 publications

References 8 publications

Harnessing the Power of Induced Pluripotent Stem Cells and Gene Editing Technology: Therapeutic Implications in Hematological Malignancies

Harnessing the Power of Induced Pluripotent Stem Cells and Gene Editing Technology: Therapeutic Implications in Hematological Malignancies

Induced pluripotent stem cell models of myeloid malignancies and clonal evolution

Modeling blood diseases with human induced pluripotent stem cells

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