30 Questions
Which of the following is a learning outcome for Week 12?
Evaluating sources of genetic information
What is the main focus of the lecture in Week 12?
The use of new genetic tools and applications in medicine
What is the LODOX System?
A technology for reducing X-ray dose
According to the text, what is the rate of progress in technology between 2000 and 2014?
100 years of technological advance
Which type of gene therapy involves inserting a 'normal' gene into somatic (adult) cells to alleviate or eliminate the symptoms of a disorder?
Somatic Cell Gene Therapy
What is the main reason why dominant disorders involving a gain-of-function mutation are more difficult to treat using Gene Therapy?
The patient already has enough of that gene or gene product
Which method of introducing genes into cells involves small membrane-like microspheres that contain the gene to be expressed?
Liposomes
Which type of virus is commonly used in Gene Therapy as a viral vector to introduce 'foreign' DNA into cells?
Adeno-associated virus
Which method is used to create a three-person baby by removing the pronuclei and adding the parents' pronuclei to the donor embryo?
Method 1: Embryo Repair
What is the estimated ratio of children born with diseases caused by mitochondrial mutations?
1 in 5,000
Which gene mutation is associated with Cystic fibrosis?
CFTR gene
How many hits match the given DNA sequence in the human genome?
Over 200
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?
Ex vivo gene therapy
Which gene therapy approach is useful for disorders where only one tissue is affected or causes symptoms?
In situ gene therapy
Which gene therapy product was the first one approved by the European Medicines Agency (EMA) in 2012?
Glybera
Which gene therapy product utilizes Adeno-associated virus (AAV9) to deliver functional copies of the SMN1 gene to neurons?
ZOLGENSMA
Which gene does the drug in the CRISPR clinical trial cleave?
BCL11A
What is the process that underlies haemoglobin switching?
Repression of the γ-globin gene promoter
What type of stem cells can become any cell type in the body?
Embryonic stem cells
What is the main purpose of a haematopoietic stem cell transplant?
To reset the immune system
Which RNAi-based therapy is used to treat primary hyperoxaluria type 1 (PH1)?
Lumasiran
Which RNAi-based therapy is used to treat hereditary transthyretin amyloidosis (hATTR)?
Hereditary transthyretin amyloidosis (hATTR)
What is the purpose of chromosome transplantation (CT)?
To correct cell function and development in trisomic iPS cells
What is the potential application of human artificial chromosomes (HACs)?
To treat specific genetic conditions by introducing HACs into human cells with high efficiency
According to the text, what is the purpose of educating patients to check if a clinical trial exists via ClinicalTrials.gov before agreeing?
To prevent patients from participating in clinical trials with financial conflicts of interest
According to the text, what will be the cost of getting one's own genome sequenced in less than a decade?
$100 genome
According to the text, what will personalized genome readings indicate?
The impact of genetic variation on an individual's health
According to the text, what will the future of personal genomics allow for?
All of the above
According to the text, what is the genetic component of all diseases?
Both inherited defects and environmental stresses
According to the text, what will be identified in the future for almost any trait?
Predisposition alleles (SNPs)
Test your knowledge about the CRISPR clinical trial launched in 2018 by Vertex and CRISPR Therapeutics. Learn about the drug that targets the gene BCL11A and its role in repressing the production of Fetal haemoglobin. Explore the ex vivo approach used in the trial.
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