Treatment of genetic disorders: Here's how stem cells can treat genetic diseases | Health - Hindustan Times
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Treatment of genetic disorders: Here's how stem cells can treat genetic diseases

ByZarafshan Shiraz, Delhi
Mar 21, 2023 01:01 PM IST

Health expert spills the beans on the role of cellular based therapy when it comes to genetic disorders and how stem cells can be used to treat genetic diseases

Genetic disorders are those in which genes have been altered or abolished as a contributing factor, given that both genes and the environment have a role in the development of most diseases and sometimes a single disease is brought on by a single gene deletion or mutation but they may also be inherited. A stem cell is an undifferentiated cell that splits asymmetrically and gives birth to two copies of itself together with a progenitor cell, whose progeny eventually become cells of particular tissues.

Treatment of genetic disorders: Here's how stem cells can treat genetic diseases (Photo by CDC on Unsplash)
Treatment of genetic disorders: Here's how stem cells can treat genetic diseases (Photo by CDC on Unsplash)

The stem cell now has the ability to multiply and regenerate itself. In an interview with HT Lifestyle, Dr Pradeep Mahajan, Regenerative Medicine Researcher and Founder, explained, "The majority of cell division in a repairing or regenerating tissue is accounted for by transit amplifying cells, which are frequently described in this lineage as dividing "slowly," which refers to infrequently, and producing multipotent progenitors that may divide "rapidly," which refers to frequently and with a short cell cycle. These multipotent progenitors develop into cell-specific precursors after their final division, which eventually gives rise to a mature cell type. Currently, a huge variety of cells from various stages of life are studied in relation to stem cells, including adult stem cells present in many organ tissues and embryonic stem cells found in the early blastocyst. These traits imply significant advantages over numerous suggested clinical uses for embryonic stem cells, as well as the potential for autologous transplantation."

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Highlighting that genetic disorders are already being treated with adult stem cells, he elaborated, “Haematopoietic stem cells found in the bone marrow are used in bone marrow transplants to treat inherited and acquired blood and immune system illnesses. Patients who have a recognized diagnosis and an apparent genetic mutation can donate their stem cells. These can be used, together with cells produced from them, to examine and contrast the genetic and metabolic pathways affected by the disease with cells from healthy controls. Given that both genes and the environment have a part in the development of most diseases, genetic diseases are those in which genes have been eliminated or altered as a contributing factor. Occasionally, a single gene deletion or mutation causes a single disease. They can be inherited either dominantly (where having just one copy of the faulty gene results in the disease), or recessively (in which both copies of a gene must be mutated for the disease to be manifest).”

He added, “Adult stem cells are already used to treat genetic diseases. In order to cure inherited and acquired diseases, bone marrow transplants involve haematopoietic stem cells developed in immunological and blood disorders. Individuals who have a known genetic mutation and a recognized diagnosis are eligible to donate their stem cells. They can be used to analyze and compare the genetic and metabolic pathways impacted by the disease with cells from healthy controls, along with cells made from them.”

Talking about how stem cells could be used to treat genetic diseases, Dr Pradeep Mahajan revealed, “Patients with acute leukemia, bone marrow aplasia, and congenital immunological deficiencies are treated with whole bone marrow intravenous infusions. The haematopoietic stem cell, a rare cell that serves as the basis for all adult stem cell research, possesses characteristics that are crucial to the efficacy of various treatments. However, with the advent of techniques to separate and define adult stem cells from other tissues, new cell-based therapies are being created to treat a variety of hereditary diseases, not just blood disorders.”

He suggested how genomic diseases may be treated with stem cells and said, “Individuals with congenital immunological defects, acute leukaemia, and bone marrow aplasia. Intravenous infusions of entire bone marrow are used to address deficiencies. The haematopoietic stem cell, an uncommon cell that forms the basis of all adult stem cell research, has properties that are essential to the success of numerous therapies. However, new cell-based therapies are being developed to address a variety of inherited diseases, not just blood disorders, with the development of tools to separate and identify adult stem cells from other tissues.”

Asserting that the treatment of genetic disorders is the cellular repair of tissues damaged by the genetic mutation, he shared, “Stem cells free of the mutation would be implanted to restore normal function after having been obtained from a donor who was tissue matched but had no genetic mutation. In contrast, the stem cells could come from the patient but be genetically altered before being implanted (Fischer et al. 2004). When taken from sick individuals, stem cells have the capacity to develop into the desired cell type to restore normal tissue function following transplantation. They can also serve as cellular models to study genetic and metabolic pathways in cells. When the gene acts on many organs and requires transplanting at various places, the therapeutic answer might not be evident. The most likely therapies for cellular based transplantation are those in which a single cell type may be transplanted into a single place.”

According to him, “Treatment for genetic illnesses involves cellular repair of tissues harmed by the genetic mutation, and to restore normal function, stem cells free of the mutation would be transplanted after being taken from a donor who was tissue matched but did not have the mutation. Instead, the patient's stem cells might be genetically modified before being implanted (Fischer et al. 2004). Stem cells from ill people have the capacity to differentiate into the desired cell type to re-establish normal tissue function after transplantation. Moreover, they can be used as cellular models to research the genetic and metabolic processes in cells. The therapeutic solution might not be appropriate when the gene affects many organs and calls for transplantation in different locations.”

By providing cell models for various individual diseases, adult stem cell biology has the potential to help study hereditary diseases and gene-environment interactions with stem cells, is more readily available and can be obtained from accurately diagnosed patients. and many genetic components. The health expert pointed out, “Cell models based on adult stem cells are used to determine the genes and metabolic processes involved in the cellular pathology of inherited diseases. Gene therapy and stem cell research have grown significantly. Since stem cells can self-renew, the need for repeated injections of therapeutic cells can be reduced or even eliminated with cell-based gene therapy. The main goal of gene therapy is the treatment of diseases caused by damaged genes. It achieves this by replacing the function of a defective gene with a healthy gene. This will help in the development of biomarkers and the identification of new targets for genetic-specific drug therapy.”

Dr Pradeep Mahajan concluded, “Exosomes, which transmit genetic information from cell to cell, play an important role in interactions between tumor cells and their niche, including fibroblasts, endothelial cells, adipocytes, and monocytes. Several studies have shown that tumor cells can influence their neighbouring cells by releasing exosomes. These exosomes provide signalling signals for cell stimulation, activation, proliferation, and differentiation. Exosomes contain mRNA, microRNA (miRNA), and proteins that can be transferred to target cells, causing genetic and epigenetic changes. By facilitating the horizontal transfer of bioactive molecules such as proteins, RNAs, and microRNAs, they are now thought to play important roles in tumor invasion and metastasis, inflammation, coagulation, and stem cell regeneration and expansion. The purpose of this review article is to discuss the importance of exosome-mediated cell-to-cell communication in tumor biology.”

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