Gene Therapy :: Genetics Genetic Engineering

Gene Therapy Gene therapy is the term used for the process of curing various diseases on the genetic level by injecting patients with a healthy gene so their bodies will continue to produce healthy copies of the gene rather than destructive ones. The term gene therapy and the basic notions that it entailed weren't even in researchers' vocabulary until the mid 1960's. Early endeavors to cure people via a genetic approach began in 1970 with a disease called argininemia, but proved ineffective. An actual effective transfer of a gene didn’t occur until nine years later in 1979. Early attempts to examine the ethical nature of the future of gene therapy stemmed from this initial successful experiment and ones immediately following it.2 Throughout the 1980’s and early 1990’s development of gene therapy continued. It wasn’t until 1993 that the first clinical trial of gene therapy using the adenovirus, which was the vector that Jesse Gelsinger was administered, took place.2 A vector is the method by which the healthy gene is inserted into the DNA of the patient. 18 year-old Jesse Gelsinger died September 17, 1999 from a gene therapy experiment targeting the gene for the disease ornithine transcarbamylase (OTC), a rare genetic disorder that affects the liver and its ability to clear ammonia from the body’s bloodstream. The illness occurs in 1 out of every 40,000 births and 50% of infants die within a month of birth. Jesse had a relatively mild form of the disease and was controlling it with drugs. He entered the study to help further OTC research with the hopes of finding a treatment for others with the condition.1 The study was performed by the University of Pennsylvania’s gene therapy researchers. Researchers injected Jesse’s liver with a vector including both the adenovirus and a normal OTC gene. He was the 18th subject to undergo the treatment, but received a higher dose of the adenovirus than all of the other subjects. Gene Therapy :: Genetics Genetic Engineering Gene Therapy Gene therapy is the term used for the process of curing various diseases on the genetic level by injecting patients with a healthy gene so their bodies will continue to produce healthy copies of the gene rather than destructive ones. The term gene therapy and the basic notions that it entailed weren't even in researchers' vocabulary until the mid 1960's. Early endeavors to cure people via a genetic approach began in 1970 with a disease called argininemia, but proved ineffective. An actual effective transfer of a gene didn’t occur until nine years later in 1979. Early attempts to examine the ethical nature of the future of gene therapy stemmed from this initial successful experiment and ones immediately following it.2 Throughout the 1980’s and early 1990’s development of gene therapy continued. It wasn’t until 1993 that the first clinical trial of gene therapy using the adenovirus, which was the vector that Jesse Gelsinger was administered, took place.2 A vector is the method by which the healthy gene is inserted into the DNA of the patient. 18 year-old Jesse Gelsinger died September 17, 1999 from a gene therapy experiment targeting the gene for the disease ornithine transcarbamylase (OTC), a rare genetic disorder that affects the liver and its ability to clear ammonia from the body’s bloodstream. The illness occurs in 1 out of every 40,000 births and 50% of infants die within a month of birth. Jesse had a relatively mild form of the disease and was controlling it with drugs. He entered the study to help further OTC research with the hopes of finding a treatment for others with the condition.1 The study was performed by the University of Pennsylvania’s gene therapy researchers. Researchers injected Jesse’s liver with a vector including both the adenovirus and a normal OTC gene. He was the 18th subject to undergo the treatment, but received a higher dose of the adenovirus than all of the other subjects.