Chapter CHST6, Problem 1RQ

What is gene therapy?

Summary Introduction

To review:

The meaning of the term “gene therapy.”

Introduction:

The genetic disorders are the disorders that arise due to defects in the specific genes. Various drugs are used to control the symptoms of these disorders but these drugs cannot treat the disease. To treat these genetic disorders, the technique of gene therapy is used thatcan successfully correct the genetic disorders.

Explanation of Solution

The gene therapy is a therapeutic technique that involves the insertion of one or more corrected genes into the genome of a patient suffering from a genetic disease. The corrected genes are designed in the laboratory by the experts. The expression of the corrected genes alter the DNA (deoxyribonucleic acid) or RNA (ribonucleic acid) transcript and leads to the correct protein synthesis to overcome the disorder. The vectors that are most commonly used for the introduction of modified gene in the target cell is the virus. The viruses usually attach to the host cell and insert their genetic material into it. Then, they take up the machinery of the host to replicate. These viruses can be modified to transfer only the therapeutic gene and not the viral gene. Commonly used viruses for this purpose are the adenoviruses, retroviruses, and herpes simplex virus.

The procedure of gene therapy is shown in the following figure:

Conclusion

Therefore, it can be concluded that the gene therapy is a therapeutic technique used to treat the genetic disorders by introducing the corrected genes into the cells of the patient.