scholarly journals Next Steps in Cancer Therapy: Integration of Gene Editing in Inducible Pluripotent Stem Cells

2017 ◽  
Vol 8 (1) ◽  
Author(s):  
Parastoo Boroumand ◽  
Farigol Hakem Zadeh
Keyword(s):  
Stem Cells ◽  
Cancer Therapy ◽  
Pluripotent Stem Cells ◽  
Gene Editing ◽  
Therapy Integration

scholarly journals Correction to: Gene Editing Rescues in Vitro T Cell Development of RAG2-Deficient Induced Pluripotent Stem Cells in an Artificial Thymic Organoid System

2021 ◽  
Author(s):  
Cameron L. Gardner ◽  
Mara Pavel-Dinu ◽  
Kerry Dobbs ◽  
Marita Bosticardo ◽  
Paul K. Reardon ◽  
...  
Keyword(s):  
Stem Cells ◽  
T Cell ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Gene Editing ◽  
T Cell Development ◽  
Cell Development ◽  
Induced Pluripotent

scholarly journals Gene editing and clonal isolation of human induced pluripotent stem cells using CRISPR/Cas9

Methods ◽  
2017 ◽  
Vol 121-122 ◽  
pp. 29-44 ◽  
Author(s):  
Saniye Yumlu ◽  
Jürgen Stumm ◽  
Sanum Bashir ◽  
Anne-Kathrin Dreyer ◽  
Pawel Lisowski ◽  
...  
Keyword(s):  
Stem Cells ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Gene Editing ◽  
Induced Pluripotent

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

Cells ◽  
2021 ◽  
Vol 10 (10) ◽  
pp. 2698
Author(s):  
Ishnoor Sidhu ◽  
Sonali P. Barwe ◽  
Raju K. Pillai ◽  
Anilkumar Gopalakrishnapillai
Keyword(s):  
Stem Cells ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Gene Editing ◽  
Molecular Mechanisms ◽  
Hematological Malignancies ◽  
Disease Modeling ◽  
Clonal Evolution ◽  
Induced Pluripotent ◽  
Ipsc Disease Modeling

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.

scholarly journals Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery

2020 ◽  
Vol 14 (1) ◽  
Author(s):  
Claudia De Masi ◽  
Paola Spitalieri ◽  
Michela Murdocca ◽  
Giuseppe Novelli ◽  
Federica Sangiuolo
Keyword(s):  
Stem Cells ◽  
Drug Discovery ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Gene Editing ◽  
Induced Pluripotent

scholarly journals Generation and Characterization of CPVT1 Cardiomyocytes using Human Induced Pluripotent Stem Cells and CRISPR/Cas9 Gene Editing

2018 ◽  
Vol 114 (3) ◽  
pp. 116a ◽  
Author(s):  
Naohiro Yamaguchi ◽  
Xiao-Hua Zhang ◽  
Hua Wei ◽  
Martin Morad
Keyword(s):  
Stem Cells ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Gene Editing ◽  
Induced Pluripotent

scholarly journals Silent IL2RG Gene Editing in Human Pluripotent Stem Cells

2016 ◽  
Vol 24 (3) ◽  
pp. 582-591 ◽  
Author(s):  
Li B Li ◽  
Chao Ma ◽  
Geneve Awong ◽  
Marion Kennedy ◽  
German Gornalusse ◽  
...  
Keyword(s):  
Stem Cells ◽  
Pluripotent Stem Cells ◽  
Gene Editing ◽  
Human Pluripotent Stem Cells ◽  
Il2rg Gene

scholarly journals Non-immunogenic Induced Pluripotent Stem Cells, a Promising Way Forward for Allogenic Transplantations for Neurological Disorders

2021 ◽  
Vol 2 ◽  
Author(s):  
Henriette Reventlow Frederiksen ◽  
Ulrik Doehn ◽  
Pernille Tveden-Nyborg ◽  
Kristine K. Freude
Keyword(s):  
Stem Cells ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Neurological Disorders ◽  
Developmental Disorders ◽  
Gene Editing ◽  
Current Status ◽  
Cell Replacement ◽  
Induced Pluripotent ◽  
The Brain

Neurological disorder is a general term used for diseases affecting the function of the brain and nervous system. Those include a broad range of diseases from developmental disorders (e.g., Autism) over injury related disorders (e.g., stroke and brain tumors) to age related neurodegeneration (e.g., Alzheimer's disease), affecting up to 1 billion people worldwide. For most of those disorders, no curative treatment exists leaving symptomatic treatment as the primary mean of alleviation. Human induced pluripotent stem cells (hiPSC) in combination with animal models have been instrumental to foster our understanding of underlying disease mechanisms in the brain. Of specific interest are patient derived hiPSC which allow for targeted gene editing in the cases of known mutations. Such personalized treatment would include (1) acquisition of primary cells from the patient, (2) reprogramming of those into hiPSC via non-integrative methods, (3) corrective intervention via CRISPR-Cas9 gene editing of mutations, (4) quality control to ensure successful correction and absence of off-target effects, and (5) subsequent transplantation of hiPSC or pre-differentiated precursor cells for cell replacement therapies. This would be the ideal scenario but it is time consuming and expensive. Therefore, it would be of great benefit if transplanted hiPSC could be modulated to become invisible to the recipient's immune system, avoiding graft rejection and allowing for allogenic transplantations. This review will focus on the current status of gene editing to generate non-immunogenic hiPSC and how these cells can be used to treat neurological disorders by using cell replacement therapy. By providing an overview of current limitations and challenges in stem cell replacement therapies and the treatment of neurological disorders, this review outlines how gene editing and non-immunogenic hiPSC can contribute and pave the road for new therapeutic advances. Finally, the combination of using non-immunogenic hiPSC and in vivo animal modeling will highlight the importance of models with translational value for safety efficacy testing; before embarking on human trials.

scholarly journals Modeling human neurodevelopmental diseases with brain organoids

2022 ◽  
Vol 11 (1) ◽  
Author(s):  
Xiaoxiang Lu ◽  
Jiajie Yang ◽  
Yangfei Xiang
Keyword(s):  
Stem Cells ◽  
Human Brain ◽  
Pluripotent Stem Cells ◽  
Neurodevelopmental Disorders ◽  
Human Disease ◽  
Gene Editing ◽  
Disease Modeling ◽  
Developmental Abnormalities ◽  
The Past ◽  
Underlying Mechanisms

AbstractStudying the etiology of human neurodevelopmental diseases has long been a challenging task due to the brain’s complexity and its limited accessibility. Human pluripotent stem cells (hPSCs)-derived brain organoids are capable of recapitulating various features and functionalities of the human brain, allowing the investigation of intricate pathogenesis of developmental abnormalities. Over the past years, brain organoids have facilitated identifying disease-associated phenotypes and underlying mechanisms for human neurodevelopmental diseases. Integrating with more cutting-edge technologies, particularly gene editing, brain organoids further empower human disease modeling. Here, we review the latest progress in modeling human neurodevelopmental disorders with brain organoids.

Increasing Gene Editing Efficiency for CRISPR-Cas9 by Small RNAs in Pluripotent Stem Cells

2021 ◽  
Vol 4 (4) ◽  
pp. 491-501
Author(s):  
Alireza Shahryari ◽  
Noel Moya ◽  
Johanna Siehler ◽  
Xianming Wang ◽  
Ingo Burtscher ◽  
...  
Keyword(s):  
Stem Cells ◽  
Pluripotent Stem Cells ◽  
Small Rnas ◽  
Gene Editing ◽  
Editing Efficiency

How can gene-editing of human pluripotent stem cells help cystic fibrosis?

2019 ◽  
Author(s):  
Sara Cuevas Ocana ◽  
Amy Wong ◽  
Magomet Aushev ◽  
Jin Ye Yang ◽  
Neil Perkins ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Stem Cells ◽  
Pluripotent Stem Cells ◽  
Gene Editing ◽  
Human Pluripotent Stem Cells
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