Gene Therapy—Basic Principles and the Road from Bench to Bedside

Abstract

Human gene therapy (HGT) is defined as the transfer of nucleic acids to somatic cells of a patient, which results in a therapeutic effect by correcting genetic defects, overexpressing proteins, or inhibiting the production of “harmful” proteins. In most applications, gene therapy represents a new, innovative drug delivery system making use of the technical and scientific advances of the last two decades in microbiology, virology, organic chemistry, molecular biology, biochemistry, cell biology, genetics, genomics, and genetic engineering. This article focuses on basic principles, development, and applications of gene therapy.

Future Directions in Pain Management

“…74,75 Gene Vectors: A Distinct Class of Therapeutic Agents In gene therapy, a vector serves as an intracellular delivery mechanism for the instruction set provided by the therapeutic gene (transgene) to the targeted cells. 76 The encoded proteins or peptides are expressed by the target cells, thereby harnessing their machinery to produce the therapeutic substance locally. Vectors are the most commonly genetically engineered viruses whose ability to replicate is artificially disrupted and part or all of their genome is replaced by a transgene of choice.…”