- 7 lessons
- 1 quizzes
- 10 week duration
Module 1
Introduction
Gene therapy is defined as replacing or remove the non-functional gene to a functional gene or healthy gene. It is a novel process that utilizes the sequence of gene segments as a therapeutic molecule rather than drug compounds. In this mechanism, DNA encoding a protein known as “vector” which conveys the DNA into the living host cells. The gene is replaced with minimal toxicity with the expression of enrolled DNA by the cell machinery. Most of the diseases like X-linked CID, ADA-SCID, Parkinson’s Disease, and Leukemia have noted of complete and successful clinical trials with gene delivery mechanisms. United States of America got approval in 1990 by the FDA on ADA-SCID by Gene therapy. After that, around 000 clinical trials on patients have been performed with many tools on various diseases.