Journal of Cell Science & Therapy
Open Access

Challenges of Gene Therapy and Techniques.

Zemene Kifle^{* *}Correspondence: Zemene Kifle, Department of Pharmacology, University of Gondar, College of Medicine and Health Sciences, School of Pharmacy, Gondar, Ethiopia, Email:

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About the Study

Gene therapy is a branch of medicine that focuses on genetically altering cells to provide a therapeutic effect or heal disease by repairing or reconstructing damaged genetic material.

When DNA is implanted into a patient to treat a genetic condition, this is known as gene therapy. A working gene is frequently included in the new DNA to remedy the effects of a disease-causing mutation.

Depending on the type of cell being treated, there are two types of gene therapy:

Somatic gene therapy: A piece of DNA is transferred to any bodily cell that does not make sperm or eggs. Gene therapy side effects will not be passed down to the patient's children.

Germline gene therapy: A piece of DNA is transferred to cells that create eggs or sperm. Gene therapy's effects will be passed down to the patient's children and future generations.

Gene therapy techniques

Gene therapy can be carried out using a variety of methods.

These include

Gene inhibition therapy: Infectious infections, cancer, and hereditary diseases due to abnormal gene activity can be treated with this therapy. The goal is to introduce a gene whose product either suppresses another gene's expression or interferes with the activity of another gene's product. The goal of this treatment is to shut down a gene that promotes the proliferation of diseaserelated cells.

Killing of specific cells: Suitable for disorders that can be treated by killing specific groupings of cells, such as cancer. The inserted DNA carries a "suicide" gene that causes the sick cell to die by producing a very poisonous substance. The inserted DNA promotes the expression of a protein that labels the cells,allowing the body's natural immune system to fight the sick cells. The inserted DNA must be targeted carefully with this procedure to avoid the destruction of healthy cells.

Challenges of gene therapy

Delivering the new gene to the appropriate cell and turning it on: The new gene must reach the specific cell. Delivery of a gene to the wrong cell would cause health problems and is inefficient. Another major challenge is gene turning on when it reaches a specific cell. However, cells can obstruct this process by shutting down genes that display unusual activity. The next challenge is to avoid the immunological responses. The immune system's job is to keep intruders out. New genes added through gene therapy are sometimes considered potentially hazardous intruders. The patient's immune system will react to those hazardous intruders and will try to damage them. As a result, scientists must devise a method of delivering DNA without the immune system recognizing it. This can be achieved by using vectors that are less likely to elicit an immunological response. Ensure that the new gene does not interfere with the operation of other genes: A new gene supplied through gene therapy should, in theory, integrate into the patient's genome and work for the rest of their life. There's a chance that the new gene will get in the way of another gene, causing it to stop working. It can be harmful; for example, if it interferes with a vital gene involved in cell division regulation, it could lead to cancer.

Conclusion

Although gene therapy research has progressed significantly in the last decade, practical success has yet to be demonstrated, owing to poor delivery mechanisms and gene expression after delivery. However, many geneticists believe that gene therapy will soon have its first clinical success and can treat and cure a wide range of hereditary illnesses.