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Traits are carried in DNA as instructions for constructing and operating an organism. These instructions are contained in segments of DNA called genes.

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DNA

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To provide the information needed to make proteins in cells

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Function of genes

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Genes give instructions and proteins carry out these instructions. tasks : building a new copy of a cell, or repairing damage.

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Division of labor in cells

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So it only does one job. Meaning : if a cell needs to do something new, it must make a new protein to do this job.

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Each type of protein is a specialist

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Genes tell cells what to do by telling them which proteins to make and in what amounts.

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GENES duty

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(A,T,G and C) - the sequence of nucleotides that stores information called the genetic code

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nucleotides

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When a gene is read by a cell the DNA sequence is copied into a very similar molecule called RNA (this process is called transcription).

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RNA

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Transcription is the first step of gene expression, in which a particular segment of DNA is copied into RNA by the enzyme RNA polymerase. Both RNA and DNA are nucleic acids, which use base pairs of nucleotides as a complementary language.

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Transription

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The RNA copy is then fed through the ribosome, which translates the sequence of nucleotides in the RNA into the correct sequence of amino acids and joins these amino acids together to make a complete protein chain.

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Transcription cont

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The process of moving information from language of DNA into the language of amino acids

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Translation

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A feature of an organism is called a "trait". ex. a person's eye-color, height or weight. There are many other types of traits and these range from aspects of behavior to resistance to disease.

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Traits

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For example a child might inherit the tendency to be tall, but if there is very little food where they live and they are poorly nourished, they will still be short.

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Interaction between inherited features and the environment.

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Depends on both their family history and their lifestyle.

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Chances of someone dying of heart disease and cancer

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Different forms of one type of gene are called different alleles of that gene.

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Alleles

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Mutations are random events that change the sequence of a gene and therefore create a new allele. - Mutations can produce a new trait, such as turning an allele for black hair into an allele for white hair.

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Mutations

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- Some diseases are hereditary and run in families. Other disorders are caused by a combination of hereditary and environmental factors.

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Inherited Diseases

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Caused by the environment.

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infectious diseases

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Diseases that are caused by a single allele of a gene and are inherited in families are called genetic disorders ("single-gene defects").

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Genetic disorders

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These include diseases like Huntington's disease, Cystic fibrosis, hemophilia, sickle cell anemia or Duchenne muscular dystrophy.

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Example of genetic disorders/ single gene defects

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Caused by mutations in a single gene called CFTR and is inherited as a recessive trait.

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Cystic fibrosis

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Risk of breast cancer is 50 times higher in the families most at risk compared to families least at risk.

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Breast Cancer

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due to a large number of alleles. Each of them changes the risk a little bit. - Several of the genes involved have been identified, such as BRCA1 and BRCA2, but not all of them.

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Variation of Breast cancer

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Being overweight, achohol, not excercising, increase the risk of this cancer.

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factors that increase the cause breast cancer

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The insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in which a defective mutant allele is replaced with a functional one.

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Gene therapy

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Generally, it is not an exact replacement of the "abnormal," disease-causing gene, but rather extra, correct copies of genes are provided to complement the loss of function.

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Basic process of gene therapy

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A carrier that must be used to deliver therapeutic gene to the patient's target cells. Most common types of vectors have been genetically altered to carry normal human DNA

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Vector

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Virsuses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner. - Scientists have tried to harness this ability by manipulating the viral genome to remove disease-causing genes and insert therapeutic ones.

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Virsues and genes

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Target cells such as the patient's liver or lung cells are infected with the vector.

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Steps of gene therapy + use of vectors (1)

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The vector then unloads its genetic material containing the therapeutic human gene into the target cell.

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Step (2)

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The generation of a functional protein product from the therapeutic gene restores the target cell to a normal cell.

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Step (3)

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In the case of germ line gene therapy, germ cells, i.e., sperm or eggs, are modified by the introduction of functional genes, which are ordinarily integrated into their genomes.

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Germ line therapy

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Change due to therapy would be heritable and would be passed on to later generations

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GERM LINE THERAPY CONT

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This option is prohibited for application in human beings, at least for the present, for a variety of technical and ethical reasons.

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Germ line therapy is prohibited doe

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A somatic cell is a non-sex cell.

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Somatic cells

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In the case of somatic gene therapy, therapeutic genes are transferred into the somatic cells of a patient. Any modifications and effects will be restricted to the individual patient only, and will not be inherited by the patient's offspring

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Somatic gene therapy

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A normal gene may be inserted into a nonspecific location within the genome to replace a nonfunctional gene. ( most common approach)

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1. Using viral vectors ( methods 1)

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1. Trouble preventing undesired effects. 2. Ensuring the virus will infect the correct target cell. 3. Ensuring that the inserted gene doesn't disrupt any vital genes already in the genome.

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Potential problems of using viral vectors

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An abnormal gene could be swapped for a normal gene. - This method involves a genetic exchange between two similar or identical strands of DNA). - Widely used in cells to accurately repair double-strand breaks in DNA.

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2. Homologous recombination

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Returns the gene to its normal function.

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3. Selective Reverse Mutation

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The degree to which a gene is turned on or off of a particular gene could be altered.

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4. The regulation

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Virus with a RNA genome that replicates by using a viral reverse transcriptase enzyme to transcribe its RNA into DNA in the host cell. - The virus thereafter replicates as part of the host cell's DNA

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Retroviruses

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Human immunodeficiency virus (HIV) - can lead to Acquired Immunodeficiency syndrome (AIDS)

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EXAMPLE of Retrovirus

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- One problem is that the genetic material of the virus can be inserted in any arbitrary position in the genome of the host. (The genetic material is randomly shoved into a chromosome.)

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Retroviral Vectors in Gene Therapy

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When genetic material is inserted in the middle of one of the original genes of the host cell, and the gene is be disrupted. If the gene happens to be one regulating cell division, uncontrolled cell division (i.e., cancer) can occur.

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Insertional mutagenesis

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Most successful gene therapy trails to date

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Trails using retroviral vectors to treat X-linked Severe Combined Immunodeficiency (X-SCID; "bubble-boy disease") .

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20+ patients have been treated in France and Britain, with a high rate of immune system improvement.

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Trails in Europe

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Halted or restricted in the USA when leukemia (cancer of the blood or bone marrow) was reported in patients treated in the French X-SCID gene therapy trial.

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Trails in USA

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4 children in the French trial and one in the British trial have developed leukemia as a result of insertional mutagenesis by the retroviral vector. * All but one of these children responded well to conventional anti-leukemia treatment.

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Luekemia as a result of insertional mutagenesis

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adenoid + virus; from its having first been found in adenoid tissue)

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Adenovirsuses

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- carry genetic material in the form of double-stranded DNA. - Cause respiratory, intestinal, and eye infections in humans (especially the common cold). - This vector system has shown promise in treating cancer.

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Adenovirus cont.

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Adenovirus

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1st gene therapy product to be licensed to treat cancer

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World's first gene therapy trial for inherited retinal disease.

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Milestones in Gene Therapy 2007

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- Researchers were able to give trichromatic vision1 to squirrel monkeys using gene therapy, a hopeful precursor to a treatment for colour blindness in humans. 1The ability of humans and some other animals to see different colours, mediated by interactions among three types of color-sensing cone cells in the retina. 2Primarily affects young boys; failure of the adrenal glands; progressively damages the myelin (a complex fatty neural tissue that insulates many nerves of the central and peripheral nervous systems), eventually destroying it http://vimeo.com/6987302 Worldwide, about 1,500 gene therapy trials are under way. Canada ranks fourth internationally in gene-transfer research.

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2009

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Researchers succeeded at slowing down a fatal brain disease: adrenoleukodystrophy by using a vector derived from HIV to deliver the gene to the missing enzyme.

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2009

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- failure of the adrenal glands; progressively damages the myelin (a complex fatty neural tissue that insulates many nerves of the central and peripheral nervous systems), eventually destroying it - Primarily affects young boys;

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Adrenoleukodystrophy2

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- 1,500 gene therapy trials are under way - Canada ranks 4th internationally in gene-transfer research.

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Worldwide gene therapy trails

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In 2007 and 2008, a German hematologist named Gero Hütter cured a man who had HIV infection by repeated hematopoietic stem cell transplantation with a CCR5 mutation. - Cure accepted by medical community in 2011.

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2011 milestone

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pertaining to the formation of blood or blood cells; e.g., hemopoietic stem cells in bone marrow - Some people have inherited a CCR5 mutation (called "Delta 32 mutation"). Carriers of this mutation are resistant to certain strains of HIV infection.

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hematopoietic

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- First gene therapy in the Western world to reach important regulatory approval milestone

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2012

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-Ruling by the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) that recommends marketing of a gene therapy known as Glybera (alipogene tiparvovec) as a treatment for the ultra-rare genetic disease lipoprotein lipase deficiency (LPLD) under exceptional circumstances.

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July 20, 2012

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LPLD affects no more than two people per million in the general population.

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LPLD epidimeology

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People with LPLD cannot break down fat, and must manage their disease with a restricted diet. However, dietary management is difficult, and a high proportion of patients suffer life-threatening pancreatitis.

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LPDL profile

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No gene therapy has yet been approved in the U.S. (or Canada); however, the FDA has established a system that facilitates faster reporting of adverse events in human gene transfer trials and that tracks such trials that are taking place.

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Approval of gene therapy

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- Short-lived nature of gene therapy: Before gene therapy can become a permanent cure for any condition, the therapeutic DNA introduced into target cells must remain functional and the cells containing the therapeutic DNA must be long-lived and stable.

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Challenges for gene therapy 1

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Problems with integrating therapeutic DNA into the genome and the rapidly dividing nature of many cells prevent gene therapy from achieving any long-term benefits. - Patients will have to undergo multiple rounds of gene therapy.

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Challenges for gene therapy cont. 2

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The immune system's enhanced response to invaders it has seen before makes it difficult for gene therapy to be repeated in patients.

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Immune system problems - gene therapy effectiveness 3

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Viruses present a variety of potential problems to the patient —toxicity, immune and inflammatory responses, and gene control and targeting issues. - In addition, there is always the fear that the viral vector, once inside the patient, may recover its ability to cause disease.

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Viral vectors problems 4

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- Conditions that arise from mutations in a single gene are the best candidates for gene therapy. - Unfortunately, some of the most commonly occurring disorders, such as heart disease, high blood disease, Alzheimer's disease, arthritis, and diabetes, are caused by the combined effects of variations in many genes.

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Multigene or multifactorial disorders 5

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If the DNA is integrated in the wrong place in the genome, for example in a tumor suppressor gene, it could induce a tumor (e.g., X-SCID clinical trials).

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Insertional Mutagenesis 6

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Religious groups and creationists may consider the alteration of an individual's genes as tampering or corrupting God's work.

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Religious concerns

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1. Tumor is resected and placed in tissue culture 2. The growing tumor cells are genetically altered with immunostimulatory genes 3. Document vector gene expression 4. cells are reinjected subcutaneously after irradiation

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Tumor Vaccine steps

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Inflammation, autoimmune disease

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Theoretical risks of tumor vaccines

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- needed high voltage, so people came up a transistor

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Vacuum tube guest lecture

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READINGS

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Readings

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A unit of heredity that determine the phenotypes of a person. They are made up of DNA. They are coiled up and are either promoted to produce a function or turned off to stop a function.

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What is a gene?

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It is the altering of a person's genetic material to fight or prevent disease. Its placing DNA or RNA into the cell to achieve a clinical result.

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What is gene therapy?

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In one of two ways: 1- to make healthy cells fight the cancerous cells, or 2 - to target cancer cells to destroy them. Some of these approaches include improving the immune system's response to cancer, replacing missing or deformed genes with healthy ones, making the cancer cells more responsive to treatment.

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How is gene therapy being used in cancer treatment?

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Through a vector - usually a virus. It can be done ex vivo- where the insertion takes place outside the body or in vitro - inside the body

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How are genes transferred into cells so that gene therapy can take place?

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Retroviruses, adenoviruses, adeno-associated viruses, lentiviruses, proxy viruses, and herpes viruses. - They differ in the ability to cause permanent or temporary change, and their ability to recognize and infect certain target cells. - They are safe because they have been genetically altered to not produce disease, but still carry out other functions.

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What type of viruses are used in gene therapy, and how can they be used safely?

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- That the viruses may infect more than the target cell type, or insert it into an improper location causing mutation. - It could also insert it into germ cells in the body (sperm/egg), it could cause over exposure of the target gene. - The virus can cause inflammation if the immune system recognizes it, it can be pass on person to person as well.

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What risks are associated with current gene therapy trials?

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The issue of implantation of the genes- it needs to be more precise. There needs to be a better way of delivering the genes.

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What major problems must scientists overcome before gene therapy becomes a common technique for treating disease?

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- A single gene disease that makes one immune-compromised. - It was chosen because it was controlled by a single gene, the regulations for that gene were simple, and the concentration in the body both high and low did not cause many problems for people.

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The first disease approved for treatment with gene therapy was adenosine deaminase (ADA) deficiency. What is this disease and why was it selected?

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They must be approved by at least 2 review boards, and by the FDA separately

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How do gene therapy trials receive approval?

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It could lead to many problems if it's not done correctly

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Why are there so many steps in this process?

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Germ cell therapy - is an ethical issue. Social issues - are good because treatment for diseases may come about.

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What are some of the social and ethical issues surrounding human gene therapy?

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Theres a research program dedicated to this called the ethical, social and legal research program that deals with the social and ethical implications of the human genome project.

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What is being done to address these social and ethical issues

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1. The problem of clinical utility and relative risk-given the multifactorial nature of common disease and the weak predictive properties of genetic-risk alleles, the probability of misclassifying individuals as high or low is too high of a risk for it to be useful today.

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ii. Deflating the Genomic Bubble. • Describe 4 substantial impediments to realizing many of the claims of genomics.

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The illusion of parsing risk- defining relative risk on the basis of individuals' genetics is less meaningful when the absolute risk for everyone is high.

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2.

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The difficulty of changing behaviours - knowing about a higher risk for a certain disease does not make people take meaningful actions to stop said behaviour.

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3.

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The paradox of risk information - if genetic information tells one person that they are genetically susceptible to a disease, then there's another person who isn't and that would lead to both of them changing behaviours. It could lead to counterproductive behaviours.

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4.

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