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GENE THERAPY QUIZ

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Gene therapy
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what is the experimental technique for correcting the defective genes responsible for disease development
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human gene therapy
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use of genes as medicines or drugs to treat or prevent human disease by correcting altered genes in patient’s bodies to rid them of illness forever
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Adenosine deaminase (ADA) deficiency
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what deficiency is caused by Severe Combined Immune Deficiency (SCID)
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SCID
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disorders that cause severe abnormalities in the immune system by reducing the numbers and/or function of T and B lymphocytes
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it is difficult or impossible for the body to battle the viruses, bacteria, and fungal infections
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what happens when the immune system doesn’t function properly?
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specialized white blood cells made in bone marrow and the thymus gland that fight infection
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lymphocytes
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chromosome 20
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the gene on what chromosome is mutated in ADA
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breaks down a toxic substance called deoxyadenosine.
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what does ADA do?
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deoxyadenosine
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what toxin builds up and destroys infection-fighting immune cells (T&B lymphocytes)
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1. replacing missing or mutated genes that cause disease with healthy copies 2. introducing a new gene into the body to help fight a disease 3. changing the regulation of a gene
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3 different approaches to gene therapy
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replacing missing or mutated genes
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most common approach to gene therapy
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USA USA USA
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what country performs 63.7 % of the word’s clinical trials in gene therapy
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1990
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when was the first therapeutic human gene therapy clinical trial approved?
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CANCER 64%
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what disease is the most common studied in clinical trials of gene therapy
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inherited single gene (monogenetic disorders)
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these diseases are what types of disorders -severe combined immunodeficiency -cystic fibrosis -duchenne muscular dystrophy -familial hypercholesterolemia
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hematopoietic cells
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what is mainly afffected by SCID
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airway epithelia
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what is mainly affected by cystic fibrosis
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liver
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what is mainly affected by familial hypercholesterolemia
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acquired disorders (cancer)
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disorders that involve mutation in oncogenes, tumor suppressor genes and DNA repair genes
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CTFR gene (chr. 7)
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what gene is mutated that causes cystic fibrosis
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germ-line gene therapy
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therapy that involves transfer of genes to human embryonic cells; creation of transgenic humans
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gene transfer ti embryonic cells is successfully practiced in animals; this is not primarily for therapeutic purposes, but for creating recombinant proteins *targets germ cells, which allows the inserted gene to be passed to futute generations; NOT practiced in humans for ethical reasons
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features of germ line gene therapy
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somatic cell gene therapy
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therapy that involves transfer of genes to somatic cells in order to correct or replace the function of affected genes
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may be introduced to somatic cells at all levels of development *NO impact on future generations as changes to somatic cells are not heritable
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features of somatic cell gene therapy
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objectives of somatic cell gene therapy
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which type of therapy’s main objective is to cure hereditary acquired genetic defects by insertion of normal genes to target cells and to restore the normal functioning if affected cells in the body
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somatic gene therapy; somatic
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which type of therapy has no effect on future generations as changes to the ____cells cannot be inherited
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PROCESS OF GENE THERAPY
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1. cells are removed from the body 2. in the lab, a virus is altered so that it cannot reproduce 3. a gene is inserted into the virus 4. the altered virus is mixed with cells from the patient 5. the cells from the patient become genetically altered 6. the altered cells are returned to the body 7. the altered cells produce the desired protein
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when it is relatively easy to take the cells from the patients body and propagate them in sufficient #s outside the body
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when is ex vivo therapy possible?
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EX VIVO
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-cells are removed from the body -purified or enriched cell populations are obtained -cells are transfected with a vector -transfected cells are selected and returned to the body
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IN VIVO
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-vector is injected directly into the bloodstream or placed directly into the target tissue -adenoviral infection of tracheal epithelial or infection of a tumor mass
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-cells are derived from the patients body -involves the isolation of totipotent hematopoietic stem cells -effective in diseases which respond to bone marrow transplantation -transfection of stem cells may lead to a permanent cure
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Autologous ex vivo gene therapy
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*uses autologous cells *little chance of immune rejection since cells are taken from and infused back into the same individual *stem cells are present in low abundance, and are difficult to isolate and culture in vitro *minimal number of available promoter-enhancers sequences that can drive long-term expression of various proteins
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Pros and Cons of Autologous ex vivo gene therapy
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NON-AUTOLOGOUS EX VIVO GENE THERAPY
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gene therapy: -involves isolating cells that grow well in culture and which can be transfected efficiently, these may originate from animals or humans -after genetic modification, cells are encapsulated in an articficial semi-permeable membrane, allowing for the diffusion of therapeutic protein but bypassing the immune response
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fibroblasts, keratinocytes, astrocytes, hepatocytes, or myoblasts
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cells used for NON-AUTOLOGOUS EX VIVO GENE THERAPY
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advanced inoperable pancreatic carcinoma
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microencapsulated CYP2B1-transfected cell mediated treatment treats what? and what phase of clinical trials is it?
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patient’s own body; animal or human (not from patient)
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cells used in autologous? non-autologous?
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slight chance, high chance
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differences in immune rejection in autologous? non-autologous?
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low abundance- difficult to isolate and culture in vitro? *high abundance
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availability of autologous? non-autologous?
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lymphocytes & bone marrow stem cells–> ADA
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ADA deficiency target cells and therapeutic genes
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lung epithelial cells; CFTR
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Cystic fibrosis target cells and therapeutic genes
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liver cells; LDLR
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familial hypercholesterolemia target cells and therapeutic genes
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muscle stem cells; dystrophin
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duchenne’s muscular dystrophy target cells and therapeutic genes
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liver cells, skin cells; factor 9 gene
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hemophilia B target cells and therapeutic genes
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tumor-infiltrating lymphocytes; TNF, HLA B7
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melanoma target cells and therapeutic genes
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lung cancer cells, p53
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lung cancer target cells and therapeutic genes
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brain tumor cells, HSV-tk
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brain tumors target cells and therapeutic genes
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joints; Interleukin-1
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arthritis target cells and therapeutic genes
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lymphocytes; HSV-tk
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AIDS target cells and therapeutic genes
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gene augmentation therapy
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way to correct non-specific insertion into the genome
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correction of the target gene by mutation
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selective reverse mutation by homologous recombination
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direct killing of disease cells
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delivery of suicidal genees
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assisted killing of disease cells by immune system cells
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delivery of immune-enhancing genes
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targeted inhibition of gene expression
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regulation of gene expression
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monogenetic disease and cancer
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2 applications of gene therapy
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steps involved in ADA gene therapy
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1. harvest of BM 2. ex vivo purification (CDm34+cell) 3. cytokine cocktail+gene induction (replacement of the ADA gene) 4. gene modified CD34+cells 5. intravenous injection
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gene therapy approaches in cancer
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-replace missing or altered genes -improve immune response -introduce “suicide genes” into cancer cells -delivery of genes into cancer cells -delivery of antisense DNA and ribozymes
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1) delivery of cytokine genes into cancer cells to induce a local inflammatory reaction 2) enhance the recognition and reaction of T lymphocytes towards tumor cells by insertion of T cell receptor genes
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ways to improve immune response to cancer
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the bystander effect
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what is the transport of triphosphorylated ganciclovir between cells helps even non-transfected tumor cells
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Suicide gene therapy in vivo for brain tumors
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-retrovirus is engineered to produce and deliver herpes simplex virus thymidine kinase -the kinase phosphorylates the drug ganciclovir, which then incorporates into the DNA of dividing tumor cells and terminates chain elongation
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only infect dividing cells so the infect tumor cells but not normal brain issue
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what type of cells do retroviruses infect, what does that lead to?
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1- MRI guided stereotactic implantation of vector producer cells into CNS tumors 2-vector producing cells inside the tumor 3-retroviruses infect tumor cells 4-GCV kills infected cells
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steps in in vivo gene therapy for brain tumors
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A) adenoviral vector
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which of the following is a preferred vector of transfering gene to nerve cells? A) adenoviral vector B)HSV vector and SV40 C) retroviral vector D) vaccine vector E) none
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C) retroviral vector
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which of the following is a preferred vector of transfering gene to brain tumor cells? A) adenoviral vector B)HSV vector and SV40 C) retroviral vector D) vaccine vector E) none
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C)
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which of the following is not a preferred vector of transfering gene to nerve cells? A) adenoviral vector B)HSV vector and SV40 C) retroviral vector D) vaccine vector E) none
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A
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which of the following is not a preferred vector of transfering gene to bain tumor cells? A) adenoviral vector B)HSV vector and SV40 C) retroviral vector D) vaccine vector E) none
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virus vectors
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what type of vector is most commonly used for gene therapy
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high viral titres site-specific integration low toxicity ability to be regulated convenience and reproducibility of production
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properties of an ideal viral vector?
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no
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does an ideal viral vector exist?
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viral vectors for gene delivery
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what is used when -genes responsible for the pathogenicity of the viral vector are disabled -exploits the natural ability of the virus to infect human cells
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gene therpay with retroviral vectors
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gene therapy that 1) production of recombinant retrovirus carrying the therapeutic gene in a packaging cell line 2) integration of the therapeutic gene into the genome of human target cells using recombinant vectors
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Retroviral Vector Gene Therapy
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gene therapy with the following properties: -stable integration of therapeutic genome -minimal risk (non-pathogenic)
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MuLV
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retrovirus used commonly, that can incorporate large genes up to 7-7.5Kb
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lentiviruses
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virus that is non-pathogenic to humans, but can transduce post-miotic cells and tissue, including neurons, retinal, muscle and hematopoietic
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retrovirus, adenovirus, adeno-associated virus, HSV
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current types of viral vectors
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adeno-associated viruses
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what virus is not known to treat any symptoms
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strong inflammatory/ immune responses
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what occurs when E1 and E3 deletions happen in vectors
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retroviruses- random insertional mutagenesis adenoviruses- in vivo
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what virus(es) have stable integration? if so in what situation?
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adenovirus
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what virus has the largest insertion capacity (35kb)
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False, because only somatic cells cannot be passed on to future generations
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True or false somatic and germ line cells are used for gene therapy. Why?
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Adenoviral vectors
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which viral vector is described by the following? -vector most widely used for in vivo gene therapy -can hold up to 35kb -infect a wide variety–> high efficiency -can infect dividing and non-dividing -have high titres -and do not incorporate into the genome
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retroviruses; lentiviruses
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what virus does not infect non-dividing cells? what is one exception?
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Non-viral vectors
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what do the following have in common? -naked DNA injection -use of liposomes -receptor-mediated endocytosis
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adenoviruses
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What virus has a high immune response
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low transfection efficiency and simple large scale production and low host immunogenicity
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2 factors of non-viral vectors
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-trichromatic vision given to monkeys -gene therapy for inherited blindness -killer T cells to target cancer cells for metastatic melanoma -tumor suppressing genes in lipid based nanoparticles to reduce human lung cancer tumors in mice
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4 examples of recent developments in gene therapy research
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-small capacity for therapeutic DNa -safety concerns -low transfection efficiency
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What are 3 limitations of viral vectors
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-development of injectable vectors -targeting of vectors to specific cell populations -safe and efficient gene transfer into appropriate regions of the genome -regulation of gene expression by either administration of simple small molecule drugs or by the body’s own physiological signals -cost effective to manufacture -curing the disease
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Goals of Gene Therapy Research
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the ability of the immune system to recognize cancer cells and kill them
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what does allovectin-7 increase
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how to get the new or replacement genes into the desired tissues
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what is the main problem of gene delivery?
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phase #1 (59.6%)
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What phase of gene therapy clinical trials tests safety and efficacy in small # of humans
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combined effect in various genes
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are the following disorders caused by one singular gene or by multiple genes? -cancer, heart disease, high BP, Alzheimer’s. arthritis, and diabetes
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allovectin-7
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what substance was used to study gene therapy agent in the treatment of cancer
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plasmid/lipid complex containing the DNA sequences encoding HLA-B7 and B2 microglobulin (part of MHC I)
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what is allovectin-7 used for?
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single genes
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are single or multiple genes the best candidate for gene therapy
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-insertion mutagenesis -immune response -problems with viral vectors -affects reproductive cells -short lived nature of gene therapy -multigene disorders
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what factors keep gene therapy from becoming an effective treatment?
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insertion mutagenesis
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what causes difficulty in delivering large sections of DNA to the correct site on the comparatively large human genome
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problems with viral vectors
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what causes toxicity, immune and inflammatory responses, gene control and targeting issues, reversion of the virus to its original form
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multigenetic or multifactorial disorders
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what type of disorders are hard to treat effectively using gene therapy
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DNA
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nucleic acid that stores genetic blueprints, there are 3 billion nucleotide base pairs in the human genome
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gene
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sequence of DNA on the chromosome which codes for protein
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allele
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different forms of genes
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genotype
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inherited instructions carried within an organism’s genetic code
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-somatic gene therapy vs. germ line -who will have access to the highly expensive therapies -should it only be used for medical purposes (i.e. genetic modification of hair follicles or increased muscle mass)
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ethical issues surround gene therapy
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NO
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is gene therapy safe?
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phenotype
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an organisms observable characteristics or traits, such as its morphology, development, biochemical, or physiological properties or behaviors
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DNA polymorphism
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a common variation in the DNA sequence among individuals, often responsible for individual characteristics
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genome
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entire set of chromosomes in an organism, encompassing all of the genetic material in the cell >99% identical between individuals
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ApoE2; protection
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what ApoE allele is 7% frequency, what phenotype?
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ApoE3; neutral
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what ApoE allele has a 79% frequency, what phenotype?
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ApoE4; risk
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what ApoE allele has a 14% frequency, what phenotype
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SNP
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what is a single-letter nucleotide change in DNA
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1%
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in what % of the population do SNPs occurs
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false
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true or false, SNPs are considered
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pharmacogenetics
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what involves the influence of genetic variation on drug response in patients by correlating gene expression or SNPs with a drug’s efficacy and toxicity?
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development of rational means to optimize drug therapy, with respect to patients genotype to inc. efficacy w/ min. adverse affects
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what does pharmacogenetics aim to do?
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*apply genomics to solve problems in drug treatments *study of genetic variation among individuals in response to drugs -whole genome application -basis for personalized medicine
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4 features of pharmacogenomics
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genomics-whole genome application genetics- a single gene
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difference between pharmacogenomics vs. pharmacogenetics in terms of gene(s) interaction with drug
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genomics- uses both genetics- only DNA
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difference between pharmacogenomics vs. pharmacogenetics in terms of DNA vs RNA
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genomics- studies genome wide role of variation in drug response –> how genetic variability affects response genetics- drug metabolism
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difference between pharmacogenomics vs. pharmacogenetics in terms of drug affects
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used to identify which patients are more likely to respond to certain drugs. i.e. Herceptin/HER2
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application of pharmacogenomics in cancer
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response to drugs inlcuding warfarin
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application of pharmacogenomics in cardiovascular disease
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glucose-6-phosphate dehydrogenase in primaquine-sensitive blood cells
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application of pharmacogenomics in enzyme deficiency
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cytochrome P450 (CYP) genes and TPMT
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application of pharmacogenomics in drug metabolism