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.

scholarly journals Brain organoids and insights on human evolution

F1000Research ◽  
2019 ◽  
Vol 8 ◽  
pp. 760 ◽  
Author(s):  
Alysson R. Muotri
Keyword(s):  
Stem Cells ◽  
Human Brain ◽  
Human Health ◽  
Human Evolution ◽  
Pluripotent Stem Cells ◽  
Disease Modeling ◽  
State Of The Art ◽  
Future Perspectives ◽  
Promising Technique ◽  
Current State

Human brain organoids, generated from pluripotent stem cells, have emerged as a promising technique for modeling early stages of human neurodevelopment in controlled laboratory conditions. Although the applications for disease modeling in a dish have become routine, the use of these brain organoids as evolutionary tools is only now getting momentum. Here, we will review the current state of the art on the use of brain organoids from different species and the molecular and cellular insights generated from these studies. Besides, we will discuss how this model might be beneficial for human health and the limitations and future perspectives of this technology.

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 The application of patient-derived induced pluripotent stem cells for modeling and treatment of Alzheimer’s disease

2019 ◽  
Vol 5 (1) ◽  
pp. 21-40 ◽  
Author(s):  
Fabin Han ◽  
Chuanguo Liu ◽  
Jin Huang ◽  
Juanli Chen ◽  
Chuanfei Wei ◽  
...  
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Stem Cells ◽  
Human Brain ◽  
Induced Pluripotent Stem Cells ◽  
Pluripotent Stem Cells ◽  
Disease Modeling ◽  
Autologous Transplantation ◽  
Human Ipscs ◽  
Induced Pluripotent

Alzheimer’s disease (AD) is the most prevalent age-related neurodegenerative disease which is mainly caused by aggregated protein plaques in degenerating neurons of the brain. These aggregated protein plaques are mainly consisting of amyloid β (Aβ) fibrils and neurofibrillary tangles (NFTs) of phosphorylated tau protein. Even though the transgenic murine models can recapitulate some of the AD phenotypes, they are not the human cell models of AD. Recent breakthrough in somatic cell reprogramming made it available to use induced pluripotent stem cells (iPSCs) for patientspecific disease modeling and autologous transplantation therapy. Human iPSCs provide alternative ways to obtain specific human brain cells of AD patients to study the molecular mechanisms and therapeutic approaches for familial and sporadic forms of AD. After differentiation into neuronal cells, iPSCs have enabled the investigation of the complex aetiology and timescale over which AD develops in human brain. Here, we first go over the pathological process of and transgenic models of AD. Then we discuss the application of iPSC for disease model and cell transplantation. At last the challenges and future applications of iPSCs for AD will be summarized to propose cell-based approaches for the treatment of this devastating disorder.

scholarly journals MicroRNAs and Induced Pluripotent Stem Cells for Human Disease Mouse Modeling

2012 ◽  
Vol 2012 ◽  
pp. 1-7 ◽  
Author(s):  
Chingiz Underbayev ◽  
Siddha Kasar ◽  
Yao Yuan ◽  
Elizabeth Raveche
Keyword(s):  
Stem Cells ◽  
Mouse Models ◽  
Pluripotent Stem Cells ◽  
Human Disease ◽  
Disease Modeling ◽  
Somatic Cells ◽  
Ips Cells ◽  
Potential Candidate ◽  
Genetically Engineered Mice ◽  
Induced Pluripotent

Human disease animal models are absolutely invaluable tools for our understanding of mechanisms involved in both physiological and pathological processes. By studying various genetic abnormalities in these organisms we can get a better insight into potential candidate genes responsible for human disease development. To this point a mouse represents one of the most used and convenient species for human disease modeling. Hundreds if not thousands of inbred, congenic, and transgenic mouse models have been created and are now extensively utilized in the research labs worldwide. Importantly, pluripotent stem cells play a significant role in developing new genetically engineered mice with the desired human disease-like phenotype. Induced pluripotent stem (iPS) cells which represent reprogramming of somatic cells into pluripotent stem cells represent a significant advancement in research armament. The novel application of microRNA manipulation both in the generation of iPS cells and subsequent lineage-directed differentiation is discussed. Potential applications of induced pluripotent stem cell—a relatively new type of pluripotent stem cells—for human disease modeling by employing human iPS cells derived from normal and diseased somatic cells and iPS cells derived from mouse models of human disease may lead to uncovering of disease mechanisms and novel therapies.

Sign in / Sign up

Export Citation Format

Share Document