Cell and Gene Therapy: Pros and Cons Everyone Should Know

Overview

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• Source: Microbioz India


• Date: 11 Jul,2023

Biopharmaceutical firms are undergoing a paradigm shift as a result of ongoing developments in cell and gene therapy (CGT), which has the potential to heal certain diseases.

Both cell therapy and gene therapy are subspecialties that fall under the umbrella of the broader discipline of gene therapy. Both treatments have the goal of treating, preventing, or potentially curing diseases, and both techniques have the ability to alleviate the underlying cause of hereditary diseases as well as acquired diseases. However, the processes involved in cell and gene therapies are distinct.

 What exactly is the cell therapy?

Cell therapy, in its most basic form, refers to the practise of treating patients with the help of living cells. In point of fact, of course, it is a good deal more complicated.

Blood transfusions have been performed for approximately 100 years, and the first bone marrow transplant occurred more than 50 years ago. Both of these procedures demonstrate that the concept of transferring healthy cells to a patient in order to treat or cure a disease is not a novel one. But in the last few of decades, tremendous advancements in research surrounding stem cells have made it possible to grow and reprogram cells in order to assist the body in repairing itself by replacing damaged cells with healthy new ones.

This makes it possible to grow and reprogram cells in order to help the body repair itself. There are around 200 different types of cells that make up the human body, each of which is specialised to perform a certain function. Cells that have not yet undergone the process of differentiation might be referred to as stem cells. They are the workhorses of the body, serving as the starting point for the development of all different kinds of cell tissue. Because of this, they have an exceptionally high value.

What exactly is gene therapy and how does it work?

A malfunctioning gene and/or a mutation can be the root cause of many disorders, including haemophilia, sickle cell anaemia, and Huntington disease, to name just a few examples. The use of genetic material as a medicine is the primary focus of gene treatments, which aim to fix or replace the faulty gene function that is causing the condition.

There are a few different approaches that can be used with gene therapy. Isolating cells from a patient that have a genetic deficiency, growing these cells in a culture, adding the therapeutic gene to the cells, and then putting these cells back into the body to help fight a disease is the process that is followed in ex vivo gene therapy.

The following are some of the key distinctions between cell treatment and gene therapy:

1. The process of gene therapy includes the introduction of genetic material into a carrier or vector and the subsequent uptake of the gene into the cells that are being treated. Cell treatment is injecting the patient with cells that have been modified to perform a specific or desired function. Some treatments involve the use of gene therapy in addition to cell therapy.
2. In these treatments, stem cells are taken from the patient, genetically transformed in culture to express a new gene (usually through the use of a viral vector), increased to appropriate quantities, and then given back to the patient.
3. The purpose of cell therapy is to heal diseases by repairing or modifying certain groupings of cells, or by employing cells to transport a therapy throughout the body. Before being reintroduced into the patient’s body through cell therapy, the cells are either cultured or altered in a laboratory setting.
4. The patient’s own cells, known as autologous cells, or cells from a donor, known as allogeneic cells, may have been the source of the cells.
5. Gene therapy is an approach to the treatment of diseases that involves the replacement, inactivation, or introduction of genes into cells. This can be done either inside the body (in vivo) or outside the body (ex vivo), depending on the specific disease being treated.
6. Some treatments are classified as cell therapies as well as gene therapies. These treatments are effective because they modify genes in particular types of cells and then introduce those modified genes into the body.
7. Cell therapy is frequently utilised in the treatment of ailments like as cancer and autoimmune diseases, whereas gene therapy is predominantly utilised in the treatment of genetic problems; however, it is also being investigated for its potential in the treatment of cancer and other diseases.
8. The benefits of cell therapy might be long-term or short-term, whereas the benefits of gene therapy are normally intended to be long-term in nature.
9. Despite these distinctions, it is important to keep in mind that certain treatments can be categorised as either cell therapies or gene therapies. For instance, given that ex vivo gene therapies require the insertion of modified cells, one may classify these procedures as a form of cell therapy.