Breakthroughs
Many scientific advances must take place before our invention can be created. A lot of these have to do with unsolved problems of gene therapy. In the present, gene therapy is being researched by many scientists and organizations such as Harvard Medical School, in the Department of Genetics. Some specific drawbacks to gene therapy that are being experienced by the graduate students at Harvard are Transvection, Homolog Pairing, Structural Heterozygosity, loss of heterozygosity, and ultra-conserved elements . The first problem is transvection, or the pairing of homologous chromosomal regions. In transvection chromatin can be topologically constrained because during transvection chromosome move around. This can cause changes in gene expression and therefore change the outcome of the gene therapy. Another problem is Homolog pairing. This too, has a great influence on gene expression. These both have to do with chromosome positioning, and the Harvard Medical School is currently engaged in a search of what genes are involved in chromosomal positioning (primarily homolog pairing) they have achieved some success in this area, in locating and identifying Topoisomerase II as being important in pairing in Drosophila and also found genes in the Polycomb group that may be involved. They are also investigating how sensitive genomes in different species are to structural heterozygosity. Structural Heterozygosity is having dissimilar pairs of genes for any hereditary characteristic. The drawback relating to this is that, based on an experiment involving fungi and genome responses to the insertion and deletion of genes, it is unknown how small a difference in the chromosome structure a genome can detect. They have been researching and studies in fungi of a phenomenon called meiotic silencing of unpaired DNA (MSUD)indicates deletion and insertion of certain genes may cause a response of the genomes in germ-line 42 and 43. The loss of heterozygosity is one of the mechanisms that cause diploid cells to function as haploid cells. Some of the other causes are X-inactivation, parental imprinting, and mononallelism. Another problem is the mystery of ultra conserved elements. Since the level of conservation of these elements is high, it is easy to make the assumption that the elements have important functions. However, there is increasing evidence that at least some of these elements would create no consequence at all if deleted from the genetic sequence of a mouse. These elements have been in the human genome for millions of years for no reason and now they are taking up valuable space that could be used for life saving gene therapy.
There are also problems in other areas of our invention that need to be improved. One problem is that the retrovirus that carries our gene to the cell would have to implant the gene into a cell in the bloodstream, and these synthetic virus are not yet capable of directing themselves to a specific cell. Hopefully, if it is injected correctly, the retrovirus will be able to find its way to a cell in the bloodstream. Another problem would be that there is a slight chance that the protein could be active at the time a sickness strikes the patient. If a person who has a AA Protein in them and it is currently stopping an allergic reaction then the compliment system, which helps your body fight diseases, will not be able to protect you. These reasons and other are why scientists have not created our invention yet. Hopefully in the future scientist will be able to fix these problems with new technology to make proteins such as ours and to observe chromosomes, and betterunderstanding of genes, their functions, and other body processes.
There are also problems in other areas of our invention that need to be improved. One problem is that the retrovirus that carries our gene to the cell would have to implant the gene into a cell in the bloodstream, and these synthetic virus are not yet capable of directing themselves to a specific cell. Hopefully, if it is injected correctly, the retrovirus will be able to find its way to a cell in the bloodstream. Another problem would be that there is a slight chance that the protein could be active at the time a sickness strikes the patient. If a person who has a AA Protein in them and it is currently stopping an allergic reaction then the compliment system, which helps your body fight diseases, will not be able to protect you. These reasons and other are why scientists have not created our invention yet. Hopefully in the future scientist will be able to fix these problems with new technology to make proteins such as ours and to observe chromosomes, and betterunderstanding of genes, their functions, and other body processes.