Congenital Muscular Dystrophy diseases are serious and progressive diseases that affect the life and quality of patients. They limit the patients’ lives. Recent developments in gene therapy and the introduction of personalized treatments have been a revolution in this regard. A new era has been opened, especially in recent years.
In fact, the Nobel Prize in Chemistry in 2020 was awarded to Emmanuelle Charpentier and Jennifer A. Doudna, who did research on this subject and found the CRISPR / Cas9 technique that paved the way in gene therapy;
What is gene therapy?
All or a part of a gene in the human body may be congenitally defective, or a gene may change and mutate throughout life. These variations disrupt the process of making proteins in the human body and can cause a disease. Scientists, depending on the problem; They can change a disease-causing gene, add genes to help the body fight or treat disease, or stop genes that cause problems.
Gene therapy research is very costly studies consisting of phases that require careful study of safety and efficacy. However, many gene therapy studies for congenital muscular dystrophy diseases are continuing rapidly, especially by using new techniques discovered in recent years, and they are excited and very promising.
These treatment studies are aimed at stopping the current disease, turning it into a mild form or slowing it down.