scholarly journals Stem Cells and Gene Therapy in Progressive Hearing Loss: the State of the Art

2021 ◽  
Author(s):  
Aida Nourbakhsh ◽  
Brett M. Colbert ◽  
Eric Nisenbaum ◽  
Aziz El-Amraoui ◽  
Derek M. Dykxhoorn ◽  
...  
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Hearing Loss ◽  
Treatment Modalities ◽  
Versus Gene ◽  
Vitro Model ◽  
New Treatment ◽  
Induced Pluripotent ◽  
Therapy Strategies

AbstractProgressive non-syndromic sensorineural hearing loss (PNSHL) is the most common cause of sensory impairment, affecting more than a third of individuals over the age of 65. PNSHL includes noise-induced hearing loss (NIHL) and inherited forms of deafness, among which is delayed-onset autosomal dominant hearing loss (AD PNSHL). PNSHL is a prime candidate for genetic therapies due to the fact that PNSHL has been studied extensively, and there is a potentially wide window between identification of the disorder and the onset of hearing loss. Several gene therapy strategies exist that show potential for targeting PNSHL, including viral and non-viral approaches, and gene editing versus gene-modulating approaches. To fully explore the potential of these therapy strategies, a faithful in vitro model of the human inner ear is needed. Such models may come from induced pluripotent stem cells (iPSCs). The development of new treatment modalities by combining iPSC modeling with novel and innovative gene therapy approaches will pave the way for future applications leading to improved quality of life for many affected individuals and their families.

scholarly journals Use of organoids to study regenerative responses to intestinal damage

2019 ◽  
Vol 317 (6) ◽  
pp. G845-G852 ◽  
Author(s):  
Sarah E. Blutt ◽  
Ophir D. Klein ◽  
Mark Donowitz ◽  
Noah Shroyer ◽  
Chandan Guha ◽  
...  
Keyword(s):  
Stem Cells ◽  
Parasitic Infection ◽  
Genetically Modify ◽  
Intestinal Wall ◽  
Intestinal Damage ◽  
Therapeutic Modalities ◽  
Epithelial Regeneration ◽  
Vitro Model ◽  
Induced Pluripotent

Intestinal organoid cultures provide an in vitro model system for studying pathways and mechanisms involved in epithelial damage and repair. Derived from either embryonic or induced pluripotent stem cells or adult intestinal stem cells or tissues, these self-organizing, multicellular structures contain polarized mature cells that recapitulate both the physiology and heterogeneity of the intestinal epithelium. These cultures provide a cutting-edge technology for defining regenerative pathways that are induced following radiation or chemical damage, which directly target the cycling intestinal stem cell, or damage resulting from viral, bacterial, or parasitic infection of the epithelium. Novel signaling pathways or biological mechanisms identified from organoid studies that mediate regeneration of the epithelium following damage are likely to be important targets of preventive or therapeutic modalities to mitigate intestinal injury. The evolution of these cultures to include more components of the intestinal wall and the ability to genetically modify them are key components for defining the mechanisms that modulate epithelial regeneration.

scholarly journals Chromosomal Abnormalities in Embryonic and Somatic Stem Cells

2015 ◽  
Vol 147 (1) ◽  
pp. 1-9 ◽  
Author(s):  
Paola Rebuzzini ◽  
Maurizio Zuccotti ◽  
Carlo A. Redi ◽  
Silvia Garagna
Keyword(s):  
Stem Cells ◽  
Large Scale ◽  
In Vitro Model ◽  
Disease Modeling ◽  
Chromosomal Abnormalities ◽  
Toxicological Studies ◽  
Vitro Model ◽  
Induced Pluripotent ◽  
Potential Use

The potential use of stem cells (SCs) for tissue engineering, regenerative medicine, disease modeling, toxicological studies, drug delivery, and as in vitro model for the study of basic developmental processes implies large-scale in vitro culture. Here, after a brief description of the main techniques used for karyotype analysis, we will give a detailed overview of the chromosome abnormalities described in pluripotent (embryonic and induced pluripotent SCs) and somatic SCs, and the possible causes of their origin during culture.

Cardiomyocyte culture — an update on the in vitro cardiovascular model and future challenges

2013 ◽  
Vol 91 (12) ◽  
pp. 985-998 ◽  
Author(s):  
Sreejit Parameswaran ◽  
Sujeet Kumar ◽  
Rama Shanker Verma ◽  
Rajendra K. Sharma
Keyword(s):  
Stem Cells ◽  
Cell Biology ◽  
Embryonic Stem ◽  
Small Animal ◽  
Heart Tissue ◽  
Cellular Level ◽  
Future Challenges ◽  
Vitro Model ◽  
Induced Pluripotent

The success of any work with isolated cardiomyocytes depends on the reproducibility of cell isolation, because the cells do not divide. To date, there is no suitable in vitro model to study human adult cardiac cell biology. Although embryonic stem cells and induced pluripotent stem cells are able to differentiate into cardiomyocytes in vitro, the efficiency of this process is low. Isolation and expansion of human cardiomyocyte progenitor cells from cardiac surgical waste or, alternatively, from fetal heart tissue is another option. However, to overcome various issues related to human tissue usage, especially ethical concerns, researchers use large- and small-animal models to study cardiac pathophysiology. A simple model to study the changes at the cellular level is cultures of cardiomyocytes. Although primary murine cardiomyocyte cultures have their own advantages and drawbacks, alternative strategies have been developed in the last two decades to minimise animal usage and interspecies differences. This review discusses the use of freshly isolated murine cardiomyocytes and cardiomyocyte alternatives for use in cardiac disease models and other related studies.

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