Originators of Gene Therapy
Gene therapy began in 1972 when Friedmann and Roblin wrote a scientific paper called “Gene therapy for Human Genetic Disease?” but another man, Rogers was cited in their paper for his proposition that “exogenous good DNA” can be used to replace the malfunctioning DNA in people suffering from genetic disorders. They were the first to write about gene therapy and distribute the knowledge of gene therapy to others. In 1992 Doctor Claudio Bordignon performed the first procedure of gene therapy by using hematopoietic stem cells as vector to correct genetic disorders [11]. The work of Doctor Bordignon allowed future doctors to develop gene therapy by using vectors to cure genetic disorders [11]. Without conducting this procedure, gene therapy may not exist today. The main issue when using viral vectors for the transportation of genes is immune rejection [11]. The most commonly used vectors are viruses, but once the virus enters the body, the body’s immune system operates and attacks the cells that received the new gene [11]. In May of 2006, a team of scientists led by Dr. Luigi Naldini and Dr. Brian Brown were able to develop a way to prevent immune rejection [11]. They used a new network of genes to selectively turn off the identity of the therapeutic gene which would prevent the gene from being found and I identified by the immune system [11]. When tested on mice, the cells that received this new type of gene were not rejected. This work had an immense impact on gene therapy because now, researchers will be able to conduct and test gene therapy with different genes on different diseases without worrying about the body rejecting the genes [11]. This eventually led to the discovery of multiple uses of gene therapy on many genetic disorders [11].
What gave a kick start to gene therapy was the discovery of gene screening. Gene screening allows a doctor to identify the genetic mutations that are the causes of genetic diseases [10]. This means that scientists were able to identify the specific gene mutation that is responsible for a genetic disorder [10]. For example, cystic fibrosis is the result of a mutation in the CFTR gene on chromosome 7 [2]. When the mutation is identified, the scientist would know which gene needs to be fixed or replaced and is able to insert the specific gene needed into vectors to be carried into the body. Without finding where the mutation is, a scientist would not even be able to start gene therapy since they would not know what gene they need to replace to allow the cell to function properly. Therefore being able to identify the mutated gene was a huge leap forward in advancing gene therapy technology.
What gave a kick start to gene therapy was the discovery of gene screening. Gene screening allows a doctor to identify the genetic mutations that are the causes of genetic diseases [10]. This means that scientists were able to identify the specific gene mutation that is responsible for a genetic disorder [10]. For example, cystic fibrosis is the result of a mutation in the CFTR gene on chromosome 7 [2]. When the mutation is identified, the scientist would know which gene needs to be fixed or replaced and is able to insert the specific gene needed into vectors to be carried into the body. Without finding where the mutation is, a scientist would not even be able to start gene therapy since they would not know what gene they need to replace to allow the cell to function properly. Therefore being able to identify the mutated gene was a huge leap forward in advancing gene therapy technology.