genoterápiában

Genoterápia, a treatment modality, aims to modify a person's genes to treat or cure disease. This involves introducing genetic material into cells to compensate for abnormal genes or to make a beneficial protein. There are several approaches to gene therapy, including replacing a mutated gene that causes disease with a healthy copy, inactivating or "knocking out" a mutated gene that is functioning improperly, or introducing a new gene into the body to help fight a disease.

The genetic material is typically delivered using a vector, which is often a modified virus that has been engineered to be harmless. These viral vectors can be designed to target specific types of cells, ensuring the therapeutic gene reaches the intended location. Non-viral methods of gene delivery are also being explored, which may offer advantages in terms of safety and manufacturing.

Gene therapy is a complex field with significant potential for treating a wide range of genetic disorders, cancers, and other diseases. Research and clinical trials are ongoing to refine delivery methods, improve safety profiles, and expand the applications of this innovative therapeutic approach. While promising, gene therapy also faces challenges related to efficacy, long-term effects, and ethical considerations.