Gene Therapy

With the advancement of medical science, several treatments have been discovered to treat deadly diseases. However, there are a few diseases that are genetic and as a result are not easily curable. In a few such cases, application of gene therapy has been found to be useful. 

Gene Therapy Definition

It is an experimental technique through which healthy genes are inserted into an individual or embryo to treat disease. 

Gene therapy paves ways to replace faulty or mutated genes with new ones. 

To know how to define gene therapy, continue reading this article.

How Does Gene Therapy Works?

There are several ways through which gene therapy works. 

  • Replace a mutated gene with a healthy version of that gene. 

  • Introduce a new functioning gene to fight disease.

  • Inactivate a faulty gene that is causing disease. 

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Fig: Process of gene therapy

This gene therapy diagram shows that first, the defective genes are spotted. Then, medical experts use healthy genes to replace the faulty ones. Finally, the new gene restores the functionality of the existing cells. 

Some portions of DNA containing useful proteins enter the cells through the vectors. Inside the cell, DNA/genes start making useful proteins. After some time, the damaged cells heal and remove the source of disease.  

Next segment focuses on the varied application of gene therapy. 

Different Kinds of Gene Therapy

Primarily there are two types of gene therapy. 

  • Somatic Gene Therapy 

Human body mainly consists of somatic or stem cells. This process uses healthy genes to replace damaged ones. The therapy targets the defected cells of an individual who is suffering from a disease. Somatic cells are mainly non-reproductive. That means the effects of this therapy will not transfer to the future generation. Hence it is considered to be one of the safest applications of gene therapy. 

  • Germline Gene Therapy 

This therapy targets the germ cells of the body that produce eggs or sperms. Germline gene therapy process includes the infusion of functional DNA into cells. However, the effect of this therapy can affect future generations. Therefore the usage of this therapy is restricted in many places. For example, the European Union does not allow this process. 

Gene Therapy Application

With time, the popularity of this therapy is increasing. The application of gene therapy includes. 

  • Effectively cures several genetic disorders. 

  • Treats diseases like brain tumours, Alzheimer’s, Parkinson’s, Haemophilia and several others. 

  • Useful for the diseases that traditional medicine cannot cure.  

  • Solely destroys disease-causing cells without affecting other cells. 

  • Can be used on individuals, as well as embryos. 

However, this treatment has some temporal or permanent side effects too. 

Do It Yourself: Make a detailed note on the harmful effects of gene therapy. 

Challenges of Gene Therapy 

  • The new genes have to reach the right place. 

  • On reaching the exact location, this gene has to start becoming functional. 

  • The genes can cause harm if it reaches the wrong cells. 

  • Sometimes targeted cells stop the new genes from entering. The immune system of a body often also tries to kill the inserted gene. 

  • It has to make sure that the new cells are affecting the functions of other cells. 

In short, gene therapy can be an excellent treatment option if used properly. 

Multiple Choice Questions

1. Somatic Cell Therapy Involves

  1. Ex vivo

  2. In vivo

  3. Antisense

  4. All of these

2. Which One of these Causes from a Mutation in a Single Gene?

  1. HIV

  2. Colon cancer

  3. Cystic Fibrosis

  4. Diabetes

3. Which One Helps in Treating Cancer?

  1. In vivo

  2. Ex vivo

  3. Antisense

  4. All of the above

4. ADA Deficiency in Children can be Treated by

  1. Bone marrow transplantation

  2. Gene therapy

  3. Chemotherapy

  4. Antisense therapy

Answers: 1-d), 2-c), 3-c), 4-a)

For gene therapy project pdf for class 12, you can download our Vedantu app which allows you convenient access not just to these related study materials, but also that of online interactive sessions.

FAQ (Frequently Asked Questions)

1. What Diseases can Gene Therapy Cure?

Ans. Several genetic diseases can be treated by gene therapy. A few examples are haemophilia, cancer, diabetes, AIDS and others. 

2. Who is the Father of Gene Therapy?

Ans. William French Anderson is the father of gene therapy. 

3. How are Viruses Used in Gene Therapy?

Ans. Primarily a few viruses like retroviruses are used as vectors in gene therapy as they can infect the cells. However, viruses can work after modification so that they do not cause other diseases.