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 (B)</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 (C)</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 (C)</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 (A)</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 (B)</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 (D)</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 (A)</p> Signup and view all the answers

Which gene mutation is associated with Cystic fibrosis?

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

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

<p>Over 200 (B)</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 (A)</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 (D)</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 (B)</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 (B)</p> Signup and view all the answers

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

<p>BCL11A (B)</p> Signup and view all the answers

What is the process that underlies haemoglobin switching?

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

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

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

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

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

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

<p>Lumasiran (B)</p> Signup and view all the answers

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

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

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G&H Week 12 Lecture Notes PDF

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