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.
Gene Therapy for Diseases. Gene Therapy has made important medical advances in less than two decades. Within this short time span, it has moved from the conceptual stage to technology development and laboratory research to clinical translational trials for a variety of deadly diseases.Among the most notable advancements are the following:eurodegenerative Diseases Recent progress in gene therapy has allowed for novel treatments of neurodegenerative diseases such as Parkinson's Disease and Huntington's Disease, for which exciting treatment results have been obtained in appropriate animal models of the corresponding human diseases.