CRISPR
What is aim of CRISPR?
The aim is to treat and prevent genetic disease using this technology to allow scientists to edit genomes
True or False: DNA cloning takes the actual DNA from the nucleoid and inserts it into the bacteria
False, it inserts the plasmid into the bacteria
What is in vivo gene therapy?
It is a form of gene therapy where the cells are altered in the body
True or False: Ex vivo gene therapy is where cells are altered without the patient's consent
False
What is Gene Therapy?
The transplantation of normal genes into cells in place of missing or defective ones in order to correct genetic disorders
What does CRISPR stand for?
Clustered Regularly Interspaced Short Palindromic Repeats
What are some ethical concerns on DNA cloning
It includes: The safety and efficacy of the procedure, cloning for destructive embryonic stem cell research, the effects of reproductive cloning on the child/parent relationship
What is the advantage of having in vivo gene therapy over ex vivo gene therapy?
It avoids the cumbersome (and costly) process of removing cells from the patient, manipulating the cells in vitro, and returning the genetically modified cells to the patient.
Which of the following shows ex vivo gene therapy?
a. Cells are taken out into the sun and exposed to vitamin D to promote growth and then placed back into the patient's brain and the brain sends signals of the gene to the rest of the body
b. Cells from the patient's blood or bone marrow are removed and grown in the laboratory. The cells are exposed to the virus and it inserts desired gene and then is returned to the patient by injection into a vein.
c. Involved direct introduction of vector into the patient or near the target organ.
B
True or False: The main line of work for gene therapists is working on medical research
True
A specific type of CRISPR is the CRISPR-Cas9 system. Make an educated guess on what role Cas-9 plays in this.
Cas-9 is an enzyme that acts as a pair of 'molecular scissors' that can cut the two strands of DNA at a specific location in the genome so that bits of DNA can then be added or removed
What are the steps of DNA Cloning?
1. Cut open the plasmid and "paste" in the gene. This process relies on restriction enzymes (which cut DNA) and DNA ligase (which joins DNA).
2. Insert the plasmid into bacteria. Use antibiotic selection to identify the bacteria that took up the plasmid.
3. Grow up lots of plasmid-carrying bacteria and use them as "factories" to make the protein. Harvest the protein from the bacteria and purify it.
True or False: In vivo gene therapy is more successful than ex vivo gene therapy?
TRICK
It is not possible to determine which strategy has had more clinical success. Each strategy has had notable clinical success.
What are the 3 main steps of ex vivo?
1. Cells are taken from the patient
2. Gene is modified in the lab
3. Cells are transferred back into the patient
What is an ethical concern for gene therapy?
It may create a future where children have their genetic profiles altered in vitro so that a specific result is created. This would create different “standards” in humanity, creating a different class of person who has been “perfected.”
(answers may vary)