Transcript Leq: what is cloning and how is it done?

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-isGenomics.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/physicianresources/medical-science/genetics-molecularmedicine/current-topics/proteomics.page