Gene therapy - Mayo Clinic
网页2024年4月23日 · Gene therapy aims to fix a faulty gene or replace it with a healthy gene to try to cure disease or make the body better able to fight disease. It holds promise as a treatment for a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.
How does gene therapy work? - MedlinePlus
网页Gene therapy works by altering the genetic code to recover the functions of critical proteins. Proteins are the workhorses of the cell and the structural basis of the body’s tissues. The instructions for making proteins are carried in a person’s genetic code, and variants (or mutations) in this code can impact the production or function of ...
Gene Therapy Definition, Pros & Cons - Cleveland Clinic
网页2023年12月4日 · Gene therapy works by replacing or inactivating disease-causing genes. In some cases, gene therapy introduces new genes into your body to treat a specific disease. With gene therapy, healthcare providers deliver a healthy copy of a gene to cells inside your body.
Gene Therapy Basics | ASGCT - American Society of Gene & Cell ...
网页2022年10月31日 · Gene therapy is the use of genetic material to treat or prevent disease. The genetic material that is delivered, DNA or RNA, has instructions to change how a protein—or group of proteins—is produced by the cell.
How Does Gene Therapy Work? Types, Uses, Safety - Healthline
网页2023年1月21日 · Gene therapy can treat certain genetic conditions by editing diseased cells. Learn about its uses, safety, and potential downsides here.
What is Gene Therapy? | FDA - U.S. Food and Drug Administration
网页Gene therapies can work by several mechanisms: Replacing a disease-causing gene with a healthy copy of the gene. Inactivating a disease-causing gene that is not functioning properly....
Gene therapy | Description, Uses, Examples, & Safety Issues
网页2024年9月17日 · Gene therapy, introduction of a normal gene into an individual’s genome in order to repair a mutation that causes a genetic disease. Human gene therapy has been attempted on somatic (body) cells for diseases such as cystic fibrosis and cancer.