A5064181562- Pluripotent Stem Cells Research
- MicroRNA in disease regulation
- RNA Interference and Gene Delivery
- CRISPR and Genetic Engineering
- RNA regulation and disease
- Insect Resistance and Genetics
- Cancer-related molecular mechanisms research
- Protease and Inhibitor Mechanisms
- RNA modifications and cancer
- Signaling Pathways in Disease
- Liver physiology and pathology
Medizinische Hochschule Hannover
2016-2021
Center for Experimental and Clinical Infection Research
2021
Universidade de São Paulo
2015-2016
Recent evidence has shown that deregulated expression of members the micro RNA ‐29 (miR‐29) family may play a critical role in human cancer, including hematological malignancies. However, roles miR‐29 molecular pathophysiology T‐cell acute lymphoblastic leukemia (T‐ ALL ) not been investigated. Here, we show lower levels miR‐29a were significantly associated with higher blast counts bone marrow and increased disease‐free survival T‐ patients. Furthermore, are extremely reduced cells compared...
Abstract Homology directed repair (HDR)-based genome editing via selectable long flanking arm donors can be hampered by local transgene silencing at transcriptionally silent loci. Here, we report efficient bi-allelic modification of a locus in patient-derived hiPSC using Cas9 nickase and silencing-resistant donor construct that contains an excisable selection/counter-selection cassette. To identify the most active single guide RNA (sgRNA)/nickase combinations, employed lentiviral...
Somatic cell reprogramming by transcription factors and other modifiers such as microRNAs has opened broad avenues for the study of developmental processes, fate determination, interplay molecular mechanisms in signaling pathways. However, many that drive nuclear itself remain yet to be elucidated. Here, we analyzed role miR-29 during more detail. Therefore, evaluated expression fibroblasts transduced with lentiviral OKS OKSM vectors show addition c-MYC factor cocktail decreases levels....
Abstract The pig is an important model organism for biomedical research, mainly due to its extensive genetic, physiological and anatomical similarities with humans. Until date, direct conversion of somatic cells into hepatocyte-like (iHeps) has only been achieved in rodents human cells. Here, we employed lentiviral vectors screen a panel 12 hepatic transcription factors (TF) their potential convert porcine fibroblasts We demonstrate the first time, by over-expression CEBPα , FOXA1 HNF4α2...
Abstract Certain point-mutations in the human SERPINA1 -gene can cause severe α1-antitrypsin-deficiency (A1AT-D). Affected individuals suffer from loss-of-function lung-disease and gain-of-function liver-disease phenotypes. However, age of onset severity clinical appearance is heterogeneous amongst carriers, suggesting involvement additional genetic environmental factors. The generation authentic A1AT-D mouse-models has been hampered by complexity mouse Serpina1 locus a model with concurrent...