by Gene treatment is a technique that uses genes to treat or prevent disease. It works by introducing a normal gene to replace an abnormal gene or to make up for a missing functional gene that causes disease. The normal, therapeutic gene is packaged inside a delivery vehicle called a vector and delivered into a patient’s cells.
How Does Gene Therapy Work?
In gene treatment, the normal gene is inserted into a vector such as a virus which can deliver genetic material into a cell. The most common vectors are viruses that have been modified to carry normal genes and deliver them safely into human cells without causing disease themselves.
Once inside human cells, the normal therapeutic Gene Therapy provides instructions to produce proteins that are missing or defective, compensate for the abnormal proteins that cause disease, or interfere with the production of abnormal proteins responsible for disease. By fixing the underlying genetic defect, it aims to treat, cure, or prevent the disease.
Types of Gene treatment
There are different types of gene treatment based on its application:
Somatic Gene treatment: This type of gene treatment is aimed at treating diseases by altering gene expression in somatic cells, which make up the body’s tissues and organs, but are not involved in reproduction. Somatic cells and their descendants contain the altered gene.
Germline Gene treatment: This type aims at permanently altering the germline – egg and sperm cells and resulting offspring – with genetically modified nuclear transfer. Any changes would be passed on through future generations. However, germline therapy is currently banned in most countries due to safety and ethical concerns.
Ex vivo Gene treatment: In this method, genes are inserted into cells outside the body, such as skin or blood, in a laboratory before being returned to the patient’s body to treat disease. This allows gene transfer and monitoring of gene expression before cells are returned.
In vivo Gene treatment: In this direct method, therapeutic Gene Therapy are directly administered or delivered into a patient’s tissues or organs inside the body to treat disease.
Conditions Treated with Gene treatment
Some conditions that have been targeted for gene treatment research and clinical trials include:
– Cancer: Using genes to enhance the immune system’s ability to detect and destroy cancer cells.
– Hemophilia: Replacing the missing or defective genes that cause bleeding disorders like hemophilia A and B.
– AIDS: Attempts are being made to use gene treatment against HIV by genetically modifying cells to resist HIV infection or by boosting the immune system.
– Heart Disease: Potential uses include stimulating blood vessel growth in heart disease or regulating heart rhythm.
– Neurodegenerative Disorders: Gene treatment is being explored as a possible treatment for conditions like Alzheimer’s, Parkinson’s, and Huntington’s disease.
– Rare Genetic Disorders: Examples include treatments for conditions like SCID (severe combined immunodeficiency), beta thalassemia, and retinal disease.
Challenges and Future of Gene treatment
Despite progress, gene treatment is still in the research and development stage. Some key challenges that must still be addressed include more efficient methods for precise gene transfer and expression, addressing safety issues like unwanted immune reactions, and developing therapies for more complex conditions. Additionally, costs are currently prohibitive for widespread application.
With continual improvements and more clinical experience, gene treatment has tremendous potential to treat currently incurable or life-threatening genetic diseases. It could soon become a mainstream intervention for conditions like cancer, heart disease, and neurological disorders. As techniques develop, gene treatment may revolutionize medicine in the decades to come.
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1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it
