Gene Therapy: Therapeutic Mechanisms and Strategies

Gene therapy is a therapeutic technique in which nucleic acid is delivered into the cells of a patient as a drug to treat disease. The fundamental strategy in gene therapy is the use of DNA which when administered, reaches the damaged cells, enters the cell, and disrupts or expresses a protein. Multiple delivery techniques can be used for DNA delivery. Recent advances in the understanding of nuclease function have enabled direct DNA editing using CRISPR and zinc finger nucleases. Genes are ...