The Cystic Fibrosis Gene

Once that is done the scientist would then introduce the protein into human cells. Gene therapy can be simply viewed as inserting bits of foreign DNA into a patient’s tissue in hope... ... middle of paper ... ... fight the disease. It is crucial that regulation be a necessary component of gene therapy research and applications. In hopes that the government can regulate and can receive this treatment, not restricting it to people that has serious genetic diseases. Gene therapy will change the field of medicine from what it is today.

Gene Therapy is the process of replacing a defective gene inside a patient¡¯s DNA with a working gene that will produce the correct gene products. The genetic diseases ¡°in which a single known gene does not function properly¡±, such as sickle cell anaemia, thalassaemia and Lesch-Nyhan syndrome, are most suitable to be treated with the gene therapy. There are two types of gene therapy in curing these diseases, patient therapy and embryo therapy. In the process of the patient therapy, the first step is identifying the defective gene and isolating a normal counterpart. ¡° To obtain correct gene action, it may be necessary to put it into the correct site on the host cell chromosome, or even to delete the defective gene¡±, and the DNA can then be replicated each time the host cell divided.

The enzymes fragment foreign DNA at specific locations depending on the base sequence (Griffiths, 1996). In order to analyze an organism’s genome a researcher will add a certain restriction enzyme to DNA. This produces small restriction fragments of DNA that vary in length. Electrophoresis is then used to separate out the various fragments of DNA. This is accomplished by subjecting the fragmented DNA to an electrical charge after it has been placed onto an agarose gel plate.

Any colony exhibiting radioactivity has a protein product able to bind to the specific antibody. These colonies can then be removed and isolated. Their inserted genes can then be removed and sequenced, giving the genetic code for the DNA responsible for a particular protein of interest.

Gene splicing involves removing a fragment of DNA containing the specific DNA sequenc... ... middle of paper ... ...cientist will have to learn how to turn on specific genes when a protein of enzyme is low, and off to avoid too much to ensure the delicate chemical makeup of our bodies is not disrupted. (Blachford, 467) Although there are many risks of gene therapy, the benefits remain to enticing to ignore. Gene therapy poses many risks, but may prove the ideal solution for countless diseases. As seen throughout the past few decades, gene therapy can cure genetically inherited diseases by introducing therapeutic genes into the body. Critics may argue that there are moral and ethical problems associated with this novel technique, but for the most part scientists realize the importance this advancement will have.

DNA hybridization is the process of combining two complementary single stranded DNA or RNA molecules; they anneal using normal base pairing reactions. This can be used to detect certain sequences by using probes. The probes contain either radioactive of illuminesent materials thus it can be detected later. The probes anneal to the complementary single stranded DNA and RNA and can be visualised later by autoradiography or chemiluminescence. This allows the detection of certain gene sequences and allowing the Cystic Fibrosis gene to be pinpointed and accurately e... ... middle of paper ... ...are then designed to specifically down regulate the gene of interest.

Gene Therapy The Human Genome Project began around 1986. The main goal of the project is to locate and sequence all genes found in human DNA. The objectives for this ambitious effort are to learn more about heredity of disease and to discover the genes that would aid in gene therapy. Advances in gene therapy strive to treat hereditary diseases and possibly eliminate disease from the genome. The definition of gene therapy is the introduction of genes into existing cells to prevent or cure a wide range of diseases (Jaroff, 1996).

This paper will explain how a defect involving the replication enzyme polymerase has lead to the genetic disorder xeroderma pigmentosum or (XP). DNA Polymerase Mutation and its Impact on Replication The process of replication is essential to DNA synthesis. Without replication DNA would not be copied and divided, therefore life would not be possible. Replication is an intricate system of checks and balances, a multi step process designed to prevent errors. Accuracy is essential for replication.

When genes are altered so that the encoded proteins are unable to carry out their normal functions, genetic disorders can result. Gene therapy is a technique for correcting defective genes responsible for disease development. Researchers may use one of several approaches for correcting faulty genes: • A normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. This approach is most common. • An abnormal gene could be swapped for a normal gene through homologous recombination.

Restriction fragment length polymorphism (RFLP) analysis is a technique in which DNA regions are digested using restriction endonucleases, and subjected to radioactive complementary DNA probes to compare the differences in DNA fragment lengths between individuals. The DNA in question is digested using restriction endonuclease(s). The DNA is then run on a gel and appears to be very long. The gel is subject to a chemical that causes the double-stranded DNA to separate into to individual strands. The strands are then transferred to a nylon membrane with using an electric current, where it will bind.