The CRISPR Clinical Trial
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Questions and Answers

Which of the following is a learning outcome for Week 12?

  • Describing the progress of technology
  • Understanding the ethical issues in genetic technologies
  • Explaining the risk in genetic analysis
  • Evaluating sources of genetic information (correct)

    • What is the main focus of the lecture in Week 12?

  • The use of new genetic tools and applications in medicine (correct)
  • The ethical issues associated with novel genetic technologies
  • The widening knowledge gap between average person and educated professionals
  • The advancements in technology and science of medicine

    • What is the LODOX System?

  • A genetic analysis tool
  • A machine for understanding the human genome
  • A medicine that affects genetic technologies
  • A technology for reducing X-ray dose (correct)

    • According to the text, what is the rate of progress in technology between 2000 and 2014?

    <p>100 years of technological advance</p> Signup and view all the answers

    Which type of gene therapy involves inserting a 'normal' gene into somatic (adult) cells to alleviate or eliminate the symptoms of a disorder?

    <p>Somatic Cell Gene Therapy</p> Signup and view all the answers

    What is the main reason why dominant disorders involving a gain-of-function mutation are more difficult to treat using Gene Therapy?

    <p>The patient already has enough of that gene or gene product</p> Signup and view all the answers

    Which method of introducing genes into cells involves small membrane-like microspheres that contain the gene to be expressed?

    <p>Liposomes</p> Signup and view all the answers

    Which type of virus is commonly used in Gene Therapy as a viral vector to introduce 'foreign' DNA into cells?

    <p>Adeno-associated virus</p> Signup and view all the answers

    Which method is used to create a three-person baby by removing the pronuclei and adding the parents' pronuclei to the donor embryo?

    <p>Method 1: Embryo Repair</p> Signup and view all the answers

    What is the estimated ratio of children born with diseases caused by mitochondrial mutations?

    <p>1 in 5,000</p> Signup and view all the answers

    Which gene mutation is associated with Cystic fibrosis?

    <p>CFTR gene</p> Signup and view all the answers

    How many hits match the given DNA sequence in the human genome?

    <p>Over 200</p> Signup and view all the answers

    Which type of gene therapy involves removing target cells from the body, culturing them in the laboratory with a vector, and re-inserting them back into the body?

    <p>Ex vivo gene therapy</p> Signup and view all the answers

    Which gene therapy approach is useful for disorders where only one tissue is affected or causes symptoms?

    <p>In situ gene therapy</p> Signup and view all the answers

    Which gene therapy product was the first one approved by the European Medicines Agency (EMA) in 2012?

    <p>Glybera</p> Signup and view all the answers

    Which gene therapy product utilizes Adeno-associated virus (AAV9) to deliver functional copies of the SMN1 gene to neurons?

    <p>ZOLGENSMA</p> Signup and view all the answers

    Which gene does the drug in the CRISPR clinical trial cleave?

    <p>BCL11A</p> Signup and view all the answers

    What is the process that underlies haemoglobin switching?

    <p>Repression of the γ-globin gene promoter</p> Signup and view all the answers

    What type of stem cells can become any cell type in the body?

    <p>Embryonic stem cells</p> Signup and view all the answers

    What is the main purpose of a haematopoietic stem cell transplant?

    <p>To reset the immune system</p> Signup and view all the answers

    Which RNAi-based therapy is used to treat primary hyperoxaluria type 1 (PH1)?

    <p>Lumasiran</p> Signup and view all the answers

    Which RNAi-based therapy is used to treat hereditary transthyretin amyloidosis (hATTR)?

    <p>Hereditary transthyretin amyloidosis (hATTR)</p> Signup and view all the answers

    What is the purpose of chromosome transplantation (CT)?

    <p>To correct cell function and development in trisomic iPS cells</p> Signup and view all the answers

    What is the potential application of human artificial chromosomes (HACs)?

    <p>To treat specific genetic conditions by introducing HACs into human cells with high efficiency</p> Signup and view all the answers

    According to the text, what is the purpose of educating patients to check if a clinical trial exists via ClinicalTrials.gov before agreeing?

    <p>To prevent patients from participating in clinical trials with financial conflicts of interest</p> Signup and view all the answers

    According to the text, what will be the cost of getting one's own genome sequenced in less than a decade?

    <p>$100 genome</p> Signup and view all the answers

    According to the text, what will personalized genome readings indicate?

    <p>The impact of genetic variation on an individual's health</p> Signup and view all the answers

    According to the text, what will the future of personal genomics allow for?

    <p>All of the above</p> Signup and view all the answers

    According to the text, what is the genetic component of all diseases?

    <p>Both inherited defects and environmental stresses</p> Signup and view all the answers

    According to the text, what will be identified in the future for almost any trait?

    <p>Predisposition alleles (SNPs)</p> Signup and view all the answers

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