Gene Knockout in Hematopoietic Stem and Progenitor Cells Followed by Granulocytic Differentiation

Mir, Perihan; Ritter, Malte; Welte, Karl; Skokowa, Julia; Klimiankou, Maksim

doi:10.1007/978-1-0716-0290-4_26

Cited by 3 publications

(2 citation statements)

References 19 publications

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“…Taken into consideration a relative rarity of CN with even less CN/AML cases, BAALC expression levels and MK2a phosphorylation status should be evaluated in a bigger cohort of CN/AML patients and iPSC models with different CNassociated mutations in, for example, HAX1, JAGN1, or SRP54. (Mir et al, 2020). Off-target sites were predicted using https://crispor.tefor.net (Concordet and Haeussler, 2018).…”

Section: Limitations Of Studymentioning

confidence: 99%

iPSC modeling of stage-specific leukemogenesis reveals BAALC as a key oncogene in severe congenital neutropenia

et al. 2021

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Section: Limitations Of Studymentioning

confidence: 99%

iPSC modeling of stage-specific leukemogenesis reveals BAALC as a key oncogene in severe congenital neutropenia

et al. 2021

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“…In a recent study, over 80% of hPSCs were shown to undergo targeted genome editing using CRISPR-Cas9 reagents and nucleofection. 104 Promising advances could emerge from these studies. The combination of these methods could lead to the generation of heritable overexpression or deletion of miRNAs in platelets differentiated from transduced hematopoietic stem cells.…”

Section: In Vitro Study Of Platelet Functionmentioning

confidence: 99%

Methods to Investigate miRNA Function: Focus on Platelet Reactivity

et al. 2020

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MicroRNAs (miRNAs) are small noncoding RNAs modulating protein production. They are key players in regulation of cell function and are considered as biomarkers in several diseases. The identification of the proteins they regulate, and their impact on cell physiology, may delineate their role as diagnostic or prognostic markers and identify new therapeutic strategies. During the last 3 decades, development of a large panel of techniques has given rise to multiple models dedicated to the study of miRNAs. Since plasma samples are easily accessible, circulating miRNAs can be studied in clinical trials. To quantify miRNAs in numerous plasma samples, the choice of extraction and purification techniques, as well as normalization procedures, are important for comparisons of miRNA levels in populations and over time. Recent advances in bioinformatics provide tools to identify putative miRNAs targets that can then be validated with dedicated assays. In vitro and in vivo approaches aim to functionally validate candidate miRNAs from correlations and to understand their impact on cellular processes. This review describes the advantages and pitfalls of the available techniques for translational research to study miRNAs with a focus on their role in regulating platelet reactivity.

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CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia

Nasri,

Ritter,

Mir

et al. 2024

Molecular Therapy

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Gene Knockout in Hematopoietic Stem and Progenitor Cells Followed by Granulocytic Differentiation

Cited by 3 publications

References 19 publications

iPSC modeling of stage-specific leukemogenesis reveals BAALC as a key oncogene in severe congenital neutropenia

iPSC modeling of stage-specific leukemogenesis reveals BAALC as a key oncogene in severe congenital neutropenia

Methods to Investigate miRNA Function: Focus on Platelet Reactivity

CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia

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