Exploring the Applications of Pluripotent Stem
Cells in Disease Modeling and Precision
Sarvananda L1* and Amal D Premarathna2
1Molecular Nutritional and Biochemistry Laboratory, University of Peradeniya, Sri Lanka
2School of Natural Sciences and Health, Tallinn University, Narva mnt 29, 10120 Tallinn, Estonia, Europe
Submission: February 17, 2023; Published: February 24, 2023
*Corresponding author:Sarvananda L, Molecular Nutritional and Biochemistry Laboratory, University of Peradeniya, Sri Lanka
How to cite this article:Sarvananda L* and Amal D Premarathna. Exploring the Applications of Pluripotent Stem Cells in Disease Modeling and Precision Medicine. J of Pharmacol & Clin Res. 2023; 9(2): 555757. DOI: 10.19080/JPCR.2023.09.555757
Pluripotent stem cells (PSCs) are cells that can differentiate into any cell type in the body, and they have the potential to revolutionize genetic analysis and disease modeling. In this essay, I will discuss how PSCs can be used to model diseases and study their genetic basis. I will also examine the advantages and limitations of this approach, as well as the ethical considerations surrounding the use of PSCs.
PSCs can be derived from a variety of sources, including embryos, adult tissues, and reprogrammed cells. These cells can then be differentiated into specific cell types, allowing researchers to create in vitro models of various diseases. For example, PSCs have been used to model neurodegenerative diseases such as Parkinson’s and Alzheimer’s, as well as genetic disorders such as cystic fibrosis and sickle cell anemia . By creating disease-specific PSC lines, researchers can study the molecular and cellular mechanisms underlying the disease in question. This can include examining the effects of specific genetic mutations, as well as testing potential drug therapies. In some cases, PSCs can also be used to generate functional tissues or organs, allowing for more physiologically relevant disease models .
In addition to disease modeling, PSCs can also be used to study the genetic basis of disease. By creating PSC lines from individuals with specific genetic mutations, researchers can examine the effects of those mutations in a controlled setting.
This can help to identify the specific genes and pathways involved in disease development, as well as potential therapeutic targets . PSCs can also be used to study the effects of epigenetic modifications on gene expression. For example, researchers can create PSC lines from individuals with epigenetic disorders such as Beckwith-Wiedemann syndrome, which is characterized by abnormal gene expression due to changes in DNA methylation patterns . By studying these cells, researchers can gain a better understanding of how epigenetic modifications affect gene expression and potentially develop new treatments for epigenetic disorders.
One of the main advantages of PSC-based disease modeling and genetic analysis is the ability to create disease-specific cell lines. This allows researchers to study the effects of specific genetic mutations or environmental factors on disease development in a controlled setting. PSCs also have the potential to generate functional tissues and organs, allowing for more physiologically relevant disease models . Another advantage of PSC-based research is the ability to generate large quantities of cells for analysis. This can be especially useful for drug discovery, as researchers can test potential therapies on a large number of cells in a relatively short amount of time. PSCs can also be used to generate cells for transplantation, potentially offering new treatments for a variety of diseases . However, there are also several limitations to PSC-based research. One major limitation is the potential for genetic and epigenetic abnormalities in PSC lines. These abnormalities can affect the differentiation potential of PSCs and may lead to inconsistencies in disease modeling and genetic analysis . Another limitation
is the potential for immune rejection when using PSC-derived
tissues for transplantation. While PSCs have the potential to
generate functional tissues and organs, there is still a long
way to go before these therapies become a reality. Researchers
must overcome the challenge of immune rejection and ensure
the safety of PSC-derived tissues before these therapies can be
widely adopted .
The use of PSCs raises several ethical considerations,
particularly in the case of embryonic stem cells (ESCs). While
ESCs have the greatest differentiation potential, their use has
been controversial due to the fact that they are typically derived
from discarded embryos from in vitro fertilization procedures.
This has led to debates over the ethical implications of using
embryos for research purposes. To address these concerns,
researchers have developed alternative sources of PSCs, such
as induced pluripotent stem cells (iPSCs), which are generated
by reprogramming adult cells . While iPSCs have some
limitations compared to ESCs, they offer a more ethically
acceptable source of PSCs for research purposes. In addition to
the ethical considerations surrounding the use of PSCs, there
are also concerns about the potential misuse of this technology.
For example, PSCs could potentially be used for reproductive
cloning or other unethical purposes. As such, there are calls
for regulation and oversight of PSC research to ensure that it is
conducted in an ethical and responsible manner .
Pluripotent stem cells have the potential to revolutionize
genetic analysis and disease modeling, offering a powerful
tool for understanding the molecular and cellular mechanisms
underlying a variety of diseases. While there are limitations and
ethical considerations surrounding the use of PSCs, researchers
are continuing to explore the potential of this technology for
both research and therapeutic applications.