Chapter 16.4, Problem 1C

Summary Introduction

To determine: The potential concerns regarding the use of viral vectors in gene therapy.

Concept introduction: Gene therapy is the process of replacing an abnormal gene with a normal copy of the gene for the normal expression of a gene. This is done by inserting a gene into an individual’s cells and ‘knocking out’ the mutated gene. Cell-based delivery of a normal gene copy in the body involves the use of stem cells, fibroblast, or lymphocyte. These cells are taken from the body and a transgene is inserted and infused in the patient.