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WorksheetsGene Therapies
Total questions: 15
Worksheet time: 13mins
Adding a corrected version of a gene or modifying a defective gene to make it work properly is also known as...
gene modifacation.
retro-viral therapy.
genetic tweaking.
genetic therapy.
A genetic disorder that primarily affects the lungs and is due to a gene mutation on chromosome 7 is...
cystic fibrosis.
fibromyalgia.
lupus.
Lou Gehrig's Disease.
Which of the following would exclude a disease from being a good candidate for gene therapy?
a disease that is due to a single nucleotide base mutation.
a disease that is well understood.
a disease where the genes involved were well known.
a disease that is caused by many genes spread over many chromosomes.
The means of delivering DNA into the genome of a host is known as a(n)...
vector.
gene carrier.
intron.
base editor.
Which of the following is NOT a viral vector?
adenovirus
adeno-associated virus
liposome
Herpes simplex
What is an advantage of using non-virus associated gene therapies like liposomes and naked DNA?
They can carry much larger DNA fragments.
They enter the cell much more easily.
They readily bind with host DNA much easier.
The DNA is always compatible integrates in the right spot.
When a therapy is conducted on tissues and cells that have been taken outside of the body and put into culture dishes, it is known as...
extra-corporeal therapy.
ex vivo therapy.
post host gene modification.
in vivo therapy.
When a therapy is conducted by injecting a vector into the body and modifications occur within the host it is known as...
extra-corporeal therapy.
ex vivo therapy.
intra host gene modification.
in vivo therapy.
Which of the following is NOT an advantage of a viral vector?
very good at entering cells.
can carry DNA fragments larger than 20,000 bp
can be programmed to selectively target certain cells or tissues.
can be modified so they don't grow out of control
Circular pieces of DNA called ____________ are used by bacteria to transmit genes to each other and they are now used to package DNA in non-viral vectors
plasmids
enterosomes
base editors
gene splicers
A ______________ is a type of cell that can differentiate into many other types of cells as it matures.
base cell
skin cell
osteocyte
stem cell
One of the major problem in developing gene therapies for rare diseases that involve just one base pair is...
it doesn't make economic sense for large pharmaceutical companies to spend the money to find a cure for so few people.
it is too difficult to find cures for many of these diseases.
single base pair mutations are the hardest to cure.
we don't have the right vectors to deliver the DNA into the nucleus.
A mutated gene that produces a protein that blocks the function of a normal protein is known as a...
gain of function protein.
improper regulation protein.
dominant negative mutation.
gene function silencer.
The use of a DNA sequence to bind to sites within the DNA to form a triple helix portion of the DNA and preventing a protein from being made is called...
gene silencing.
gene enhancement.
genetic down cycling.
a triple lindy.
Which type of vector has been used to cure skin cancers and goes by the name T-VEC treatment?
adenovirus
liposome
herpes simplex 1
HIV
