Genetic Sequence Engineering

In the disease, incorrect cistron splicing results in a hemoglobin deficit, and in the crude treatment, the high-risk RNA sequences are masked with U7 snRNA which has a complementary site for the defective splicing site on the pre- mRNA, a small molecule of nuclear RNA which ordinarily processes mRNA for histones. The splicing is do using lentivirus as a vector. Since the solitary(prenominal) cure for thalassemia is bone marrow transplantation, this masking of the defective gene splicing site offers a much necessitate treatment. It is similarly a better treatment than trying to flip-flop the gene, which has been done in the past, because that results in excess RNA and hemoglobin production, which also causes problems.

Adenoviruses throw long been used as vectors in gene therapy, but they do not get for ever so in the body, and multiple doses are not possible because the repellent system develops resistance to them (1:24). Work is right away being done on an adenovirus transposon vector that atomic number 50 stabilize transgene rule over time. In vitro, stability has been achieved for six months or more. It has also sounded in the mouse model. This technology promises to make one-time application of the therapy last a lifetime.

Parkinson's disease occurs because of a profound difference of a ad hoc type of nerve cell which lies mystifying in the brain and produces dopamine, a neurotransmitter (2:4). A reduction in dopamine levels leads to

7. American dietetic Association. Position of the American Dietetic Association: biotechnology and the future(a) of food. J. Am. Dietetic Assoc. 95:1429-1432; 1995.

Conventional gene therapy to induce rude(a) vessel growth in cardiac patients with gourmandizeed vessels or poor circulation uses injections of genes that principle for vascular endothelial growth factor, a normal protein which induces new vessel growth (4:28). Researchers have now found a way to stimulate a patient's let genes to produce more vessels. They have engineered transcription factors which act instanter on a patient's DNA to stimulate or block protein production.

The transcription factors bind to unique DNA sequences near specific medically important genes and turn protein production up or down, as required. This is preferable to traditional gene therapy because the injected genes only code for one protein, but genes often make a satisfying series of proteins, and turning the gene on allows it to function normally and make all the needed proteins for vessel growth. The work is before long being done in mice, but it is hoped it will work equally well in humans. The current transcription factors have to be delivered by traditional gene therapy methods to reach a patient's DNA, but researchers hope to make a version which can be given intravenously.

2. New study shows efficacy of gene therapy. Gene Ther. Weekly. Nov. 7:4; 2002.

6. Rifkin, J. Future pharming. Animals. 131:24-27; 1998.

It is believes that genetic engineer may allow the use of certain promising antitumor agents which are no longer used because they are catchy to come by and proved too toxic to patients in initial trials, though they were very trenchant tumor inhibitors (3:10). The antitumour agents are called maytansinoids, and were originally obtained from rare Ethiopian plants, and later were set-apart from a new bacteria species. In early tests, these maytansinoids were effective against some tumors and leukemias. R
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