Gene Therapy Essay

2620 Words 11 Pages
Gene Therapy

I. Introduction

With the human genome project now completed, identifying our DNA, the next step forward is being taken to analyze this information and apply it in a helpful context. As we discover which genes affect and trigger the different traits humans possess, new questions result pertaining to potential problems in our DNA as well as genetic enhancement opportunities. In theory, once a problem is pinpointed within a person’s DNA, there exists a possibility for correcting this defect. Gene therapy is a technique used for correcting defective genes responsible for disease development. This relatively new idea sparks much controversy when societal implications are examined. Bill Joy, chief scientist and
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The former concentrates on treating the individual, while the latter aims to eliminate “bad” genes from the individual as well as their offspring.

Somatic gene therapy up to this point has been used to treat rare, deadly, genetic diseases by the insertion of specific genes into human cells other than the cells directly used in reproduction. One problem is the method of insertion. The methods that transfer the new DNA to a defective cell are referred to as vectors. The most commonly used vectors are viruses, more specifically, adenoviruses or retroviruses ( Adams , 2004). Because of the viruses’ natural ability to get by the body’s immune system and introduce DNA into cells without being detected right away, researchers find them to be a particularly useful tool. First the virus must be stripped of its harmful properties and then forced to take up the corrective DNA. The remaining goal is successful delivery to many cells in order for replication as well as insertion into the correct place in the chromosome.

Delivery can be categorized as ex vivo or in vivo. Ex vivo, the first delivery method used, removes cells from the body in order to modify and correct the DNA. Through the aid of a vector and after cell modification, the cells are transplanted back into the patient where they will replicate. Blood or liver cells are good candidates for this method (“Gene Therapy”, 2004). In vivo
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