NIH Genetic Modification Clinical Research Information System
A product that seeks to provide genes that correct or supplant the disease-controlling functions of cells that are not, in essence, doing their job. Somatic gene therapy introduces therapeutic genes at the tissue or cellular level to treat a specific individual. Germ-line gene therapy inserts genes into reproductive cells or possibly into embryos to correct genetic defects that could be passed on to future generations. Initially conceived as an approach for treating inherited diseases, like cystic fibrosis and Huntington's disease, the scope of potential gene therapies has grown to include treatments for cancers, arthritis, and infectious diseases.
A drug's generic name is one that a non-government organization assigns to the drug's active ingredient. Different \"brand names\" can be used for the same generic name ingredient.
A non-registered user of the Portal. Guests may enter and submit a safety report in the same session; but without a system account, the system cannot save an incomplete report and recognize the user if they wish to return later.
The rights and permissions given to a non-registered user of a system or service.