Gene therapy will only work for certain ailments if we can send a normal gene to a high number of cells in a tissue like, several million. They must also be the right cells in the right tissue. To generate the protein it encodes, the gene must be activated, or switched on, after it has arrived at its target. And it has to stay on once it’s switched on, cells have a history of shutting down genes that are overly active or showing other strange characteristics. Introducing modifications to the incorrect cells. The effectiveness of any gene therapy treatment depends on the ability to target a gene to the right cells.

Challenges of gene therapy-

Now we will learn about stem cell, gene therapy, risks and challenges of gene therapy and stem cell research and lastly at what are limitations of gene therapy.

Stem cell-

A stem cell is a kind of cell that has the unique potential to differentiate into different cell types in the body. They may be utilised to replace cells and tissues that have been injured or destroyed as a result of illness in the future.

Our bodies are made up of many various kinds of cells, most of which are specialised to fulfil certain activities, such as red blood cells, which transport oxygen throughout our bodies through the bloodstream, but are unable to divide.

Stem cells help the body expand by generating new cells and replacing damaged or lost specialised cells. They can accomplish this because of two unique properties-

1. They can divide indefinitely to make new cells.

2.As they divide, they may transform into the many cell types that make up the human body.

Gene therapy-

For therapeutic reasons, gene therapy tries to modify the biological features of living cells or regulate the expression of a gene. Gene therapy is a treatment or cure for sickness that involves modifying a person’s DNA. Gene therapies may work in a number of ways:

1.Replacing a disease-causing gene with a healthy copy

2.Inactivating a disease-causing gene that isn’t functioning properly.

3.A new gene is delivered into the body to cure sickness.

Cancer, genetic ailments, and other diseases are among the conditions for which gene therapy is being investigated.

Gene therapy may be delivered in a variety of methods.

1.Plasmid DNA is a circular kind of DNA that can be genetically manipulated to deliver therapeutic genes to human cells.

2.Virus vectors are utilised in a variety of gene therapy products because of their natural ability to transport genetic information into cells. If viruses have been altered to lower their infectious disease potential, they may be used as vectors (vehicles) to carry therapeutic genes into human cells.

3.Bacterial vectors may be designed to prevent infection before being used as vectors (vehicles) to carry therapeutic genes into human tissues.

4.The goal of human gene editing technology is to either destroy or repair harmful genes.

5.Patient-derived cellular gene therapy products- Patient’s cells are extracted, their genes modified (usually with the assistance of a viral vector), and then returned.

Risks and challenges of gene therapy and stem cell research-

Risks of gene therapy- 

1.The body’s immune system may react to the newly introduced therapeutic vector as if it were an invader, just as it would to any virus. Inflammation and other hazardous consequences might result from an immune system response.

2.An immune response might render gene therapy less effective, or even ineffective. As a result, prospective gene therapy patients are often tested with a simple blood test to see whether they have antibodies to a particular virus.

3.While the goal of utilising a certain vector is to send a new gene to a specific tissue type, viruses may influence cells that aren’t targeted, causing harm or producing extra sickness or disease.

Challenges of gene therapy-

1.Security is the first of many key obstacles that gene therapy has faced. Assuring that the proper replacement gene is delivered to the required cells without causing undue harm.

2. The immunogenicity of gene therapies has caused many researchers to doubt their long-term effectiveness. All biological medications are restricted in their capacity to elicit the body’s defences, which reduces their efficacy.

Stem cell research-

Risks-

1.Mucositis (mouth inflammation or sores) is a short-term adverse effect of chemo and radiation treatments. It normally improves after a few weeks of therapy, although it may make eating and drinking very uncomfortable.

2.Vomiting and nausea

Because chemotherapy treatments may produce severe nausea and vomiting, physicians often prescribe anti-nausea medications at the same time as the chemotherapy. The objective is to avoid nausea and vomiting as much as possible, since it is simpler to prevent them than it is to stop them once they begin.

Challenges-

1.Genetic inconsistency

2.The environment in which stem cells are cultured.

3.There is a pharmacological problem.

4.The dispersion of stem cells following transplantation.

What are limitation of gene therapy-

1. Selecting the incorrect cells to target. Because viruses may infect several cell types, it’s feasible that the modified viruses will infect more cells.

2.Infection brought on by a virus. It’s conceivable that once viruses are introduced into the body, they will regain their potential to cause illness.

3.The risk of creating a tumour. There’s a danger that if the new genes are introduced in the incorrect place in your DNA, they’ll cause a tumour to emerge.

Conclusion-

In this article we have read about what we mean by gene therapy, stem cell, risks and challenges of both gene and stem cell research. Gene therapy may alter cells both within and outside the body. When done within the body, a doctor would inject a gene-carrying vector straight into the portion of the body with damaged cells, and for stem cells, it will improve knowledge of how illnesses develop. Researchers may get a better understanding of how illnesses and ailments emerge by monitoring stem cells evolve into cells in bones, heart muscle, neurons, and other organs and tissue.