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Leq : What are the benefits of DNA Technology & the Human Genome Project?. 12.13 to 12.20. Gene Therapy. Gene therapy is an experimental technique that uses genes to treat or prevent disease. Researchers are testing several approaches to gene therapy, including:
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Leq: What are the benefits of DNA Technology & the Human Genome Project? 12.13 to 12.20
Gene Therapy • Gene therapy is an experimental technique that uses genes to treat or prevent disease. Researchers are testing several approaches to gene therapy, including: • Replacing a mutated gene that causes disease with a healthy copy of the gene. • Inactivating, or “knocking out,” a mutated gene that is functioning improperly. • Introducing a new gene into the body to help fight a disease. • Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently only being tested for the treatment of diseases that have no other cures. • http://ghr.nlm.nih.gov/handbook/therapy/genetherapy
Gene Therapy A carrier molecule called a vector must be used to deliver the therapeutic gene to the patient's target cells. Currently, the most common vector is a virus that has been genetically altered to carry normal human DNA. Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner. Scientists have tried to take advantage of this capability and manipulate the virus genome to remove disease-causing genes and insert therapeutic genes. http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml
Polymerase Chain Reaction - PCR A technique used to obtain many copies of a DNA molecule or part of a DNA molecule. A small amount of DNA is mixed with DNA polymerase, DNA nucleotide, and Primers then subjected to cycles of heating and cooling to produce multiple copies of DNA.
Human Genome Project Completed in 2003, the Human Genome Project (HGP) was a 13-year project coordinated by the U.S. Department of Energy and the National Institutes of Health. During the early years of the HGP, the Wellcome Trust (U.K.) became a major partner; additional contributions came from Japan, France, Germany, China, and others. Project goals were to: identify all the approximately 20,000-25,000 genes in human DNA, determine the sequences of the 3 billion chemical base pairs that make up human DNA, store this information in databases, improve tools for data analysis, transfer related technologies to the private sector, and address the ethical, legal, and social issues (ELSI) that may arise from the project. Though the HGP is finished, analyses of the data will continue for many years http://www.ornl.gov/sci/techresources/Human_Genome/home.shtml
The Human Genome • Estimated number of human genes? • 20,000 – 25,000 • Regions that make up a human chromosome? • DNA wraps around histone proteins to condense into a chromosome • DNA regions include the following regions: promoter, enhancer, introns, exons, noncoding DNA (between genes) and repetitive DNA regions
Genomics • Genomics is the study of the genomes of organisms. The field includes intensive efforts to determine the entire DNA sequence of organisms and fine-scale genetic mapping efforts. • Genomics was established by Fred Sanger when he first sequenced the complete genomes of a virus and a mitochondrion. His group established techniques of sequencing, genome mapping, data storage, and bioinformatic analyses in the 1970-1980s. • A major branch of genomics is still concerned with sequencing the genomes of various organisms, but the knowledge of full genomes has created the possibility for the field of functional genomics, mainly concerned with patterns of gene expression during various conditions. http://www.news-medical.net/health/What-is-Genomics.aspx
Proteomics • Proteomics studies the structure and function of proteins, the principal constituents of the protoplasm of all cells. • What is a proteome?The word “proteome” is derived from PROTEins expressed by a genOME, and it refers to all the proteins produced by an organism, much like the genome is the entire set of genes. The human body may contain more than 2 million different proteins, each having different functions. As the main components of the physiological pathways of the cells, proteins serve vital functions in the body http://www.ama-assn.org/ama/pub/physician-resources/medical-science/genetics-molecular-medicine/current-topics/proteomics.page