inâ¢iâ¢tial. 1. of, pertaining to, or occurring at the beginning; first: the initial step in a process. 2. an initial letter, as of a word. 3. the first letter of a proper name. 4. a large, often ornamental letter used at the beginning of a chapter or other division of a book or manuscript. 5. to mark or sign with an initial or the initials of one's name, esp. as a token of approval. initiÄlis, derivative of initi(um) beginning, derivative of inÄ«re to enter, begin = in-in- 2 + Ä«re to go]. inâ¢iâ²tialâ¢er, n.
Why Gene Therapy Is Important. Genes that are flawed and do not work properly can cause disease. Gene therapy is a technique for correcting defective genes responsible for disease development. Researchers may use one of several approaches for correcting faulty genes: 1  A normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. This approach is most common. 2  An abnormal gene could be swapped for a normal gene through homologous recombination. The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function. The regulation (the degree to which a gene is turned on or off) of a particular gene could be altered.
If cleared by the FDA, it would be the first gene therapy approved in the United States. But unlike traditional gene therapy, the new treatment doesn't replace disease-causing genes with healthy ones. Instead, it uses technology to reprogram immune cells called T cells to target and attack malignancies. When a patient is treated under the Novartis process, T cells are extracted from a patient's blood, frozen and sent to the company's plant in Morris Plains, N.J.