PATHOLOGY

Detailed explanation-1: -Gene therapy can compensate for genetic alterations in a couple different ways. Gene transfer therapy introduces new genetic material into cells. If an altered gene causes a necessary protein to be faulty or missing, gene transfer therapy can introduce a normal copy of the gene to recover the function of the protein.

Detailed explanation-2: -Genetic therapies aim to treat or cure conditions by correcting problems in your DNA. Your DNA, including specific genes, contains instructions for making proteins that are essential for good health. Mutations, or changes in your DNA, can lead to proteins that do not work properly or that are missing altogether.

Detailed explanation-3: -Gene therapy is a technique that modifies a person’s genes to treat or cure disease. Gene therapies can work by several mechanisms: Replacing a disease-causing gene with a healthy copy of the gene.

Detailed explanation-4: -In the future, genetic therapies may be used to prevent, treat, or cure certain inherited disorders, such as cystic fibrosis, alpha-1 antitrypsin deficiency, hemophilia, beta thalassemia, and sickle cell disease. They also may be used to treat cancers or infections, including HIV.

Detailed explanation-5: -Many genetic disorders result from gene changes that are present in essentially every cell in the body. As a result, these disorders often affect many body systems, and most cannot be cured. However, approaches may be available to treat or manage some of the associated signs and symptoms.