Genetic therapies aim to treat and cure conditions relating to DNA. A person’s DNA consists of instructions for creating proteins and lipids that are good for health. When DNA gets mutated, the instructions alter, and this leads to the creation of proteins that are bad for health. DNA mutations cause many disorders such as thalassemia, cystic fibrosis, hemophilia, and sickle cell disease.
Gene therapies can treat genetic disorders by providing new DNA or correcting DNA within certain cells. Gene transfer approaches, or gene addition, replace a missing function within a gene by adding a new gene to the affected cells. The new gene is either a normal replacement of a faulty gene or a different gene that solves the problem and works to improve the cell’s overall function. Genome editing is a newer method of gene therapy that allows for precise correction in changing DNA to restore cell function.
Genome editing can remove a certain part of DNA that causes diseases, disable a gene to prevent it from producing harmful proteins, enable a gene to create beneficial proteins for the cell, or correct a mutated gene. Gene transfers or genome editing can modify the cells directly in the body, or the cells can be collected and treated outside of the body. For example, a doctor could extract faulty bone marrow cells, modify the DNA, and insert it back into the body.
The only gene editing therapies that are approved by the U.S. Food and Drug Administration (FDA) are for an inherited eye condition and certain forms of cancer. If genetic therapies were more commonly used in clinical applications, they could treat and cure any other inherited disorders, treat other cancers, and treat infections.
References
https://www.nhlbi.nih.gov/health/genetictherapies#:~:text=Genetic%20therapies%20am%20to %20treat,or%20that%20are%20missing%20altogether.
Written by Aanya Deshpande from MEDILOQUY
Is there any reason why Gene Therapy is not allowed as a form of therapy for other genetic disorders yet?