Download Slide 1 - Kenwood Academy High School

Turn in your Unit 2
Formative (rubric
stapled)
Do Now – 11.22.16






Which two vectors carry plasmid DNA?
Naked DNA and liposome
Which two vectors carry ssDNA? Adenoassociated virus and Herpes Simplex Virus
Which vector carries dsDNA? Adenovirus
Which vector carries RNA? Retrovirus
Which vector has a maximum capacity of
5,000 bp? Adeno-associated virus
Take out your 2.2.1 packet and Unit 2 Review
Guide – turn to the vector table
Do Now – 11.22.16






Which two vectors carry plasmid DNA?
Naked DNA and liposome
Which two vectors carry ssDNA? Adenoassociated virus and Herpes Simplex Virus
Which vector carries dsDNA? Adenovirus
Which vector carries RNA? Retrovirus
Which vector has a maximum capacity of
5,000 bp? Adeno-associated virus
Take out your 2.2.1 packet and Unit 2 Review
Guide – turn to the vector table
Do Now – 11.22.16






Which two vectors carry plasmid DNA?
Naked DNA and liposome
Which two vectors carry ssDNA? Adenoassociated virus and Herpes Simplex Virus
Which vector carries dsDNA? Adenovirus
Which vector carries RNA? Retrovirus
Which vector has a maximum capacity of
5,000 bp? Adeno-associated virus
Take out your 2.2.1 packet and Unit 2 Review
Guide – turn to the vector table
Do Now – 11.22.16






Which two vectors carry plasmid DNA?
Naked DNA and liposome
Which two vectors carry ssDNA? Adenoassociated virus and Herpes Simplex Virus
Which vector carries dsDNA? Adenovirus
Which vector carries RNA? Retrovirus
Which vector has a maximum capacity of
5,000 bp? Adeno-associated virus
Take out your 2.2.1 packet and Unit 2 Review
Guide – turn to the vector table
Do Now – 11.22.16






Which two vectors carry plasmid DNA?
Naked DNA and liposome
Which two vectors carry ssDNA? Adenoassociated virus and Herpes Simplex Virus
Which vector carries dsDNA? Adenovirus
Which vector carries RNA? Retrovirus
Which vector has a maximum capacity of
5,000 bp? Adeno-associated virus
Take out your 2.2.1 packet and Unit 2 Review
Guide – turn to the vector table
Do Now – 11.22.16






Which two vectors carry plasmid DNA?
Naked DNA and liposome
Which two vectors carry ssDNA? Adenoassociated virus and Herpes Simplex Virus
Which vector carries dsDNA? Adenovirus
Which vector carries RNA? Retrovirus
Which vector has a maximum capacity of
5,000 bp? Adeno-associated virus
Take out your 2.2.1 packet and Unit 2 Review
Guide – turn to the vector table
2.2.1: Gene Therapy
© 2003 University of Utah
Vector Types
http://gslc.genetics.utah.edu
How the vector
carries genetic
material
RETROVIRUS
ADENOVIRUS
In the form of RNA
rather than DNA.
Double-stranded DNA
.
The most famous is
Human Immunodeficiency
Virus (HIV), which causes
AIDS.
The common cold virus.
8, 000 base pairs
Maximum length
of DNA that can be
inserted into vector
+ Infects only
dividing cells
Permission gran
ADVANTAGES
ADENO-ASSOCIATED VIRUS
HERPES
SIMPLEX VIRUS
LIPOSOME
NAKED DNA
Single-stranded DNA
Single-stranded DNA
Plasmid DNA
packaged into
miniature lipid
–based pockets
that can fuse to
the cell’s own
membranes,
The DNA is
released and
transported into
the nucleus
Plasmid DNA molecule all by itself that
can be taken up
by some cells, and
transported into the
nucleus
7,500 base pairs
5,000 base pairs
20,000 base pairs
+ Infects both dividing
and non-dividing cells
very effectively
+ Possible to target
specific cell types by
engineering proteins
on the virus surface
to recognize special
proteins on the target
cell’s surface
+ Does not cause illness in humans
+ Infects a wide range of dividing and nondividing cell types very effectively
+ Need the assistance of a “helper” virus to
replicate themselves inside cells
+ Possible to target specific cell types by
engineering proteins on the virus surface
to recognize special proteins on the target
cell’s surface
+ Integrates into the host cell’s genome;
95% of the time, it will integrate into a
specific region on Chromosome 19, greatly
reducing the chance that integration will
disrupt the function of other genes in the
+ Infects cells of the
nervous system
+ Will not integrate
into the host cell’s
genome, but is a
circular piece of DNA
that replicates when
the cell divides; will
stay in the cell’s
nucleus for a long
time
+ Will not disrupt
the function of other
genes in the host cell
+ Will not
generate
an immune
response
+ Better suited
for ex vivo
gene therapy
approaches
+ Will not generate
an immune
response
+ Generally not
toxic
Objective
 SWBAT
choose the appropriate
vector for each gene therapy
scenario.
 SWBAT
review for their Unit 2 Test.
2.2.1: Gene Therapy
Name: ______________________________
Date: ___________________ Period: _____
Activity 2.2.1 Gene Therapy
Introduction
In Lesson 2.1, you explored the science behind genetic testing and screening. You saw how DNA
sequencing technology and bioinformatics have opened the door to identifying specific genes in our
genome. Faulty genes are often at the heart of devastating illnesses and disorders. But what if
scientists could penetrate our cells and replace a gene that is malfunctioning? With a working copy of
the gene, the proper protein could be produced and the disease could be eliminated. It may sound
like science fiction, but scientists have already opened the door to this molecular treatment known as
gene therapy.
Gene therapy is the practice of inserting functional genes into a person’s genome to replace faulty
genes. Doctors and medical researchers are working to restore function to cells that are affected by
genetic disorders. To accomplish this goal, scientists have to get the right gene to the right cells in the
right tissue. This is not an easy task. Many vectors, or delivery systems, for gene therapy are actually
viruses. The idea of using viruses to deliver treatment may sound frightening, but these tiny
pathogens can be used as an effective way to target our cells and insert new genes.
In this activity, you will investigate how gene therapy works and learn about the vectors currently
being investigated as possible transport systems for new genes. You will read current research on
gene therapy trials and gather evidence for and against this type of disease treatment. Ultimately, you
will develop a policy for regulating and controlling future gene therapy research.
2.2.1: Gene Therapy
2.2.1: Gene Therapy
2.2.1: Gene Therapy
2.2.1: Gene Therapy
2.2.1: Gene Therapy
2.2.1: Gene Therapy
2.2.1: Gene Therapy
2.2.1: Gene Therapy
1.
2.
Follow the worksheet – complete on looseleaf
(determine the appropriate vectors for each
condition – then explain which vector is the best)
Answer and discuss the 2.2..1: conc questions.
2.2.1: Gene Therapy
© 2003 University of Utah
Vector Types
http://gslc.genetics.utah.edu
How the vector
carries genetic
material
RETROVIRUS
ADENOVIRUS
In the form of RNA
rather than DNA.
Double-stranded DNA
.
The most famous is
Human Immunodeficiency
Virus (HIV), which causes
AIDS.
The common cold virus.
8, 000 base pairs
Maximum length
of DNA that can be
inserted into vector
+ Infects only
dividing cells
Permission gran
ADVANTAGES
ADENO-ASSOCIATED VIRUS
HERPES
SIMPLEX VIRUS
LIPOSOME
NAKED DNA
Single-stranded DNA
Single-stranded DNA
Plasmid DNA
packaged into
miniature lipid
–based pockets
that can fuse to
the cell’s own
membranes,
The DNA is
released and
transported into
the nucleus
Plasmid DNA molecule all by itself that
can be taken up
by some cells, and
transported into the
nucleus
7,500 base pairs
5,000 base pairs
20,000 base pairs
+ Infects both dividing
and non-dividing cells
very effectively
+ Possible to target
specific cell types by
engineering proteins
on the virus surface
to recognize special
proteins on the target
cell’s surface
+ Does not cause illness in humans
+ Infects a wide range of dividing and nondividing cell types very effectively
+ Need the assistance of a “helper” virus to
replicate themselves inside cells
+ Possible to target specific cell types by
engineering proteins on the virus surface
to recognize special proteins on the target
cell’s surface
+ Integrates into the host cell’s genome;
95% of the time, it will integrate into a
specific region on Chromosome 19, greatly
reducing the chance that integration will
disrupt the function of other genes in the
+ Infects cells of the
nervous system
+ Will not integrate
into the host cell’s
genome, but is a
circular piece of DNA
that replicates when
the cell divides; will
stay in the cell’s
nucleus for a long
time
+ Will not disrupt
the function of other
genes in the host cell
+ Will not
generate
an immune
response
+ Better suited
for ex vivo
gene therapy
approaches
+ Will not generate
an immune
response
+ Generally not
toxic
2.2.1: Gene Therapy
© 2002 University of Utah
Situation
One
NFS Co.
Genetic Science Learning Center
Situation
Two
CellMart
RETROVIRUS
ADENOVIRUS
NOT POSSIBLE
The NF1 gene is encoded by 8,454 base
pairs. The gene is
too large to fit in this
type of vector.
NOT POSSIBLE
NOT POSSIBLE
NOT POSSIBLE
The NF1 gene is encoded by 8,454 base
pairs. The gene is
too large to fit in this
type of vector.
The NF1 gene is encoded by 8,454 base
pairs. The gene is
too large to fit in this
type of vector.
Although the NF1
gene contributes to
parts of the nervous
system, the HSV will
not integrate into the
host cell’s genome.
An immune response
is possible.
POSSIBLE
The vector can be
introduced into blood
stem cells, which
divide frequently.
However, the gene
may integrate into the
host cell’s genome in
random locations and
disrupt another gene.
NOT POSSIBLE
Vector infects only
ADENOASSOCIATED
VIRUS
POSSIBLE
NOT POSSIBLE
The vector can be
introduced into blood
stem cells, which divide frequently. However, it might cause
an immune response.
The blood-clotting
gene is encoded by
7,350 base pairs.
The gene is too large
to fit in this type of
vector.
POSSIBLE
The genes of this
POSSIBLE
Vector is effective at
HERPES
SIMPLEX VIRUS
NOT POSSIBLE
Blood clotting factors are not part of
the nervous system.
They are part of the
circulatory system.
NOT POSSIBLE
Lung cells are not
LIPOSOME
NOT POSSIBLE
Vector has no cell
target specificity
NOT POSSIBLE
Vector has no cell
target specificity
NOT POSSIBLE
Vector has no cell
NAKED DNA
NOT POSSIBLE
The NF1 gene is encoded by 8,454 base
pairs. The gene is
too large to fit in this
type of vector.
NOT POSSIBLE
Vector has no cell
target specificity.
NOT POSSIBLE
Vector has no cell
2.2.1: Gene Therapy
of Utah
2030 Ea
15 NorthLearning
Center,Science
Science LearningGenetic
Genetic
© 2002 University of Utah© 2002 University
Situation
One
NFS Co.
Situation
One
Two
NFS
Co.
CellMart
Situation
Situation
Two
Three
CellMart
Oma, Inc.
RETROVIRUS
ADENOVIRUS
NOT POSSIBLE
The NF1 gene is encoded by 8,454 base
pairs. The gene is
too large to fit in this
type of vector.
NOT POSSIBLE
NOT POSSIBLE
NOT POSSIBLE
The NF1 gene is encoded by 8,454 base
pairs. The gene is
too large to fit in this
type of vector.
The NF1 gene is encoded by 8,454 base
pairs. The gene is
too large to fit in this
type of vector.
Although the NF1
gene contributes to
parts of the nervous
system, the HSV will
not integrate into the
HERPES
host
cell’s genome.
SIMPLEX
An
immune VIRUS
response
is possible.
RETROVIRUS
NOT POSSIBLE
POSSIBLE
The vector
NF1 gene
The
can isbeencoded by 8,454
base
introduced
into blood
pairs.cells,
The which
gene is
stem
too large
to fit in this
divide
frequently.
type of vector.
However,
the gene
may integrate into the
host cell’s genome in
random locations and
disrupt
another gene.
POSSIBLE
The
vector
can be
NOT
POSSIBLE
introduced
intoonly
blood
Vector
infects
stem cells,
which
dividing
cells.
Lung
dividedivide
frequently.
cells
slowly, if
However,
the gene
at
all.
may integrate into the
host cell’s genome in
random locations and
ADENOVIRUS
NOT
POSSIBLE
POSSIBLE
The vector
NF1 gene
The
can isbeencoded by 8,454
base
introduced
into blood
pairs.cells,
The which
gene isdistem
too large
to fit in Howthis
vide
frequently.
type
of
vector.
ever, it might cause
an immune response.
ADENOASSOCIATED
VIRUS
ADENOASSOCIATED
VIRUS
NOT POSSIBLE
POSSIBLE
NOT
The blood-clotting
NF1 gene is enThe
codedisby
8,454 base
gene
encoded
by
pairs. base
The gene
7,350
pairs.is
too large
t in large
this
The
genetois fitoo
type
of
vector.
to fit in this type of
vector.
HERPES
SIMPLEX VIRUS
NOT POSSIBLE
POSSIBLE
NOT
Although
the NF1
Blood
clotting
facgeneare
contributes
tors
not part ofto
parts
of the nervous
the
nervous
system.
system,
HSV
will
They arethe
part
of the
not
integrate
into
the
circulatory system.
host cell’s genome.
An immune response
is possible.
POSSIBLE
NOT POSSIBLE
NOT POSSIBLE
The vector
can be
POSSIBLE
introduced
blood
The
genes into
of this
stem cells,
which
vector
do not
in- divide frequently.
tegrate
into the However, it might
cause
genome.
Repeated
an immune would
response.
treatments
be
necessary. An immune response is
The blood-clotting
POSSIBLE
gene isisencoded
Vector
effectiveby
at
7,350 base
pairs.
entering
lung
cells,
The typically
gene is too
large
and
does
to fitcause
in thisan
type
of
not
immune
vector.
response.
Blood
facNOTclotting
POSSIBLE
tors are
notare
part
of
Lung
cells
not
the nervous
system.
part
of the nervous
They are They
part of
the
system.
are
circulatory
system.
part
of the respiratory
system
LIPOSOME
NOT POSSIBLE
Vector has no cell
target specificity
LIPOSOME
NOT POSSIBLE
POSSIBLE
NOT
Vector has
has no
no cell
cell
Vector
target specifi
specificity
city
target
NAKED DNA
NOT POSSIBLE
The NF1 gene is encoded by 8,454 base
pairs. The gene is
too large to fit in this
type of vector.
NAKED DNA
NOT POSSIBLE
POSSIBLE
NOT
The NF1
gene
is enVector
has
no cell
coded specifi
by 8,454
target
city.base
pairs. The gene is
too large to fit in this
type of vector.
NOT POSSIBLE
NOT POSSIBLE
Vector
no cell
NOT has
POSSIBLE
target specifi
Vector
has nocity
cell
target specificity
Vector
no cell
NOT has
POSSIBLE
target specifi
Vector
has nocity.
cell
target specificity.
© 2002 University of Utah
RETROVIRUS
ADENOVIRUS
ADENOASSOCIATED
VIRUS
2.2.1: Gene Therapy
Situation
One
NFS Co.
Learning
Science
84112
City, UT
Salt Lake
North 2030 East,Genetic
15Utah
2002 University
Center, of
Genetic Science©Learning
Situation
Two
CellMart
Situation
Situation
Three
One
Oma,
Inc.
NFS Co.
Situation
Situation
Four
Two
SBG,
Inc.
CellMart
NOT POSSIBLE
The NF1 gene is encoded by 8,454 base
pairs. The gene is
too large to fit in this
type of vector.
POSSIBLE
The vector can be
introduced into blood
stem cells, which
divide frequently.
However, the gene
may integrate into the
RETROVIRUS
host
cell’s genome in
random locations and
disrupt another gene.
NOT POSSIBLE
The NF1
geneonly
is enVector
infects
coded bycells.
8,454Lung
base
dividing
pairs.divide
The gene
is if
cells
slowly,
tooall.
large to fit in this
at
type of vector.
POSSIBLE
POSSIBLE
The virus
vectorinfects
can be
introduced
intobut
blood
dividing
cells,
instem cells,
tegrates
intowhich
the host
dividegenome
frequently.
cell’s
in ranHowever,
the gene
dom
locations,
which
may integrate
into the
might
disrupt another
host cell’s
genome rein
gene.
An immune
random is
locations
and
sponse
possible.
disrupt another gene.
HERPES
SIMPLEX VIRUS
NOT POSSIBLE
NOT POSSIBLE
NOT POSSIBLE
The NF1 gene is encoded by 8,454 base
pairs. The gene is
too large to fit in this
type of vector.
The NF1 gene is encoded by 8,454 base
pairs. The gene is
too large to fit in this
type of vector.
Although the NF1
gene contributes to
parts of the nervous
system, the HSV will
not integrate into the
host cell’s genome.
An immune response
is possible.
POSSIBLE
NOT POSSIBLE
The vector can be
introduced into blood
stem cells, which divide frequently. However, it might cause
an immune response.
The blood-clotting
gene is encoded by
7,350 base pairs.
The gene is too large
to fit in this type of
vector.
ADENOVIRUS
NOT
POSSIBLE
POSSIBLE
The genes
NF1 gene
is enof this
coded
by
8,454
vector do not in-base
pairs. The
tegrate
intogene
the is
too large to
fit in this
genome.
Repeated
type of vector.
treatments
would be
necessary. An immune response is
possible.
POSSIBLE
vectordoes
can not
be
The virus
introducedinto
into
blood
integrate
the
stem cells,
which
genome,
and
so, divide frequently.
Howrepeated
treatments
ever, itbe
might
cause
would
necessary.
an immune
response.
Also,
may cause
an
immune response.
ADENOASSOCIATED
VIRUS
NOT
POSSIBLE
POSSIBLE
The NF1
gene is enVector
is effective
at
coded
by
8,454
base
entering lung
cells,
pairs.
The gene
and
typically
doesis
too cause
large toanfitimmune
in this
not
type of vector.
response.
NOT
POSSIBLE
POSSIBLE
The blood-clotting
Vector
infects cells
gene
is encoded
effi
ciently,
usuallyby
7,350 baseinto
pairs.
integrates
a speThe
is too large
cifi
c,gene
non-disruptive
to fit in this
type
of
location,
and
typically
vector.
does
not cause an
immune response.
NOT POSSIBLE
LIPOSOME
NOT POSSIBLE
Vector has no cell
target specificity
NOT POSSIBLE
NAKED DNA
NOT POSSIBLE
The NF1 gene is encoded by 8,454 base
pairs. The gene is
too large to fit in this
type of vector.
NOT POSSIBLE
Blood clotting factors are not part of
the nervous system.
They are part of the
circulatory system.
Vector has no cell
target specificity
Vector has no cell
target specificity.
HERPES
SIMPLEX VIRUS
LIPOSOME
NAKED DNA
NOT POSSIBLE
Although
Lung
cellsthe
areNF1
not
gene
contributes
to
part of the nervous
parts of the
nervous
system.
They
are
system,
therespiratory
HSV will
part
of the
not integrate into the
system
host cell’s genome.
An immune response
is possible.
NOT POSSIBLE
Blood clotting
T-cells
are not facpart of
torsnervous
are not part
of
the
system.
the nervous
They
are partsystem.
of the
They aresystem.
part of the
immune
circulatory system.
NOT POSSIBLE
Vector has no cell
target specifi
specificity
city
target
NOT POSSIBLE
Vector has no cell tartarget
specifi
city
get
specifi
city.
Enters
cells less effectively
than viruses.
NOT POSSIBLE
The NF1
gene
is enVector
has
no cell
coded
by
8,454
target specificity.base
pairs. The gene is
too large to fit in this
type of vector.
NOT POSSIBLE
Vector has no cell tartarget
specifi
city.
get
specifi
city.
Enters
cells less effectively
than viruses.
© 2002 University of Utah
Situation
One
NFS Co.
RETROVIRUS
ADENOVIRUS
ADENOASSOCIATED
VIRUS
NOT POSSIBLE
The NF1 gene is encoded by 8,454 base
pairs. The gene is
too large to fit in this
type of vector.
NOT POSSIBLE
NOT POSSIBLE
NOT POSSIBLE
The NF1 gene is encoded by 8,454 base
pairs. The gene is
too large to fit in this
type of vector.
The NF1 gene is encoded by 8,454 base
pairs. The gene is
too large to fit in this
type of vector.
Although the NF1
gene contributes to
parts of the nervous
system, the HSV will
not integrate into the
host cell’s genome.
An immune response
is possible.
2.2.1: Gene Therapy
of Utah
City, UT 84112Genetic Scienc
© 2002
East, Salt Lake
2030 University
Genetic Science Learning Center, 15 North
7 of 6
Situation
Two
CellMart
Situation
Three
Oma, Inc.
POSSIBLE
The vector can be
introduced into blood
stem cells, which
divide frequently.
However, the gene
may integrate into the
host cell’s genome in
random locations and
disrupt another gene.
NOT POSSIBLE
Vector infects only
dividing cells. Lung
cells divide slowly, if
at all.
RETROVIRUS
Situation
Situation
Four
One
SBG,
Inc.
NFS Co.
NOT
POSSIBLE
POSSIBLE
The virus
NF1 gene
is enThe
infects
coded
by
8,454
dividing cells, butbase
inpairs. The
is
tegrates
intogene
the host
too large
to fit in
cell’s
genome
in this
rantype
of
vector.
dom locations, which
might disrupt another
gene. An immune response is possible.
Situation
Situation
Five
Two
CF,
Inc.
POSSIBLE
NOT POSSIBLE
The vector
canonly
be
Vector
infects
introduced
into
blood
dividing cells. Lung
stem divide
cells, which
cells
slowly, if
divide
at
all frequently.
However, the gene
POSSIBLE
NOT POSSIBLE
The vector can be
introduced into blood
stem cells, which divide frequently. However, it might cause
an immune response.
The blood-clotting
gene is encoded by
7,350 base pairs.
The gene is too large
to fit in this type of
vector.
POSSIBLE
The genes of this
vector do not integrate into the
genome. Repeated
treatments would be
ADENOVIRUS
necessary. An immune response is
possible.
NOT
POSSIBLE
POSSIBLE
The virus
NF1 gene
enThe
does is
not
coded
by
8,454
base
integrate into the
pairs. The
gene
genome,
and
so, is
too
large
to
fi
t
in this
repeated treatments
type ofbe
vector.
would
necessary.
Also, may cause an
immune response.
POSSIBLE
POSSIBLE
The vector
vector is
can
be
The
effective
introduced
blood
at
entering into
non-dividstem
cells,
ing
cells
likewhich
lung divide frequently.
Howcells,
but can cause
ever, it might cause
HERPES
SIMPLEX VIRUS
NOT POSSIBLE
Blood clotting factors are not part of
the nervous system.
They are part of the
circulatory system.
POSSIBLE
NOT POSSIBLE
Vector is effective at
entering lung cells,
and typically does
not cause an immune
response.
Lung cells are not
part of the nervous
system. They are
part of the respiratory
system
ADENOASSOCIATED
VIRUS
NOT
POSSIBLE
POSSIBLE
The NF1
genecells
is enVector
infects
coded
by
8,454
base
efficiently, usually
pairs.
The
gene
is
integrates into a spetoo
large
to
fi
t
in
this
cific, non-disruptive
type of vector.
location,
and typically
does not cause an
immune response.
NOT
POSSIBLE
POSSIBLE
The vector
blood-clotting
The
is effective
gene
is encoded
by
at
entering
non-divid7,350
base
pairs.
ing cells like lung
The gene
is too large
cells,
and typically
to fit in this type of
HERPES
SIMPLEX VIRUS
NOT POSSIBLE
POSSIBLE
NOT
Although
T-cells
arethe
notNF1
part of
gene
contributes
to
the nervous system.
parts
of
the
nervous
They are part of the
system, the
HSV will
immune
system.
not integrate into the
host cell’s genome.
An immune response
is possible.
NOT POSSIBLE
POSSIBLE
NOT
Bloodcells
clotting
Lung
are facnot
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opposing viewpoint of the articles that you were assigned. Use only the notes that you took as
your partners presented their evidence.
14. When both sides have been argued, come to a consensus as to whether gene therapy research
should be continued. As a group, write a two to three paragraph statement regarding what you
believe to be effective policy for gene therapy research. Should research continue? If so, with
what restrictions? If you decide research should not be continued, explain why and provide
possible alternatives.
2.2.1: Gene Therapy
15. Explain your group’s decision and policy to the class.
16. Use the Internet to find an article about gene therapy being used for a disease, disorder or
condition NOT discussed in this activity. Bullet key points of the article in your laboratory journal
and share your findings with the class.
Conclusion
1. How do doctors and researchers decide whether a disease is a good candidate for gene therapy?
Diseases caused by single gene mutations that cause
defective proteins in somatic cells. The researchers test
out the vector before actually trying it out on a patient or a
patient’s cell. Cells are infected with the viral vector ex
vivo (outside of the body) before an in vivo therapy is
2. tried.
What are the advantages and disadvantages of using viral vectors for gene therapy?
The advantages of using viral vectors for gene therapy
include: it can target specific cells, it can integrate into the
host’s DNA. The disadvantages: can interrupt genes upon
insertion, and it can cause an immune response.
3. What factors must be considered when choosing a vector for a gene therapy trial?
2. What are the advantages and disadvantages of using viral vectors for gene therapy?
2.2.1: Gene Therapy
3. What factors must be considered when choosing a vector for a gene therapy trial?
Gene therapy is the practice of inserting functional genes
into a person’s genome to replace faulty genes. When
choosing the appropriate vector you want to be sure the
gene of interest will fit into the vector, the vector targets
the right cells, and it should limit side effects.
4. A young boy suffers from Neurofibromatosis Type 1 (NF1), a genetic nervous system disorder that
causes tumors to grow around nerve cells. The gene for NF1, approximately 8,400 base pairs
long, is located on chromosome 11. What is the best candidate for a vector for gene therapy in
this case? Provide evidence from your research to support your choice.
The keys to solving this problem are:
1) the type of cell that is the target of the vector [nerve cell]
2) the characteristics of the target cell [nerve cells do not divide]
3) the length of the gene to be carried by the vector [8,400 bp]
Sounds like Herpes Simplex Virus would be an appropriate vector.
2.2.1: Gene Therapy
5. Many athletes and administrators fear the widespread use of gene doping – the use of gene
therapy to modify genes that improve athletic performance. Describe at least two genetic
modifications that would improve the performance of an athlete. Make sure to mention the body
system(s) affected as well as the specific goal of the therapy.
Increases strength of athletes’ muscles. Could inject a
gene that generates testosterone, growth hormone, or a
muscle-strengthening protein.
6. Do you believe gene doping should be considered cheating? Why or why not?
Opinion.
6. Do you believe gene doping should be considered cheating? Why or why not?
2.2.1: Gene Therapy
7. Why do you think testing for gene doping would be extremely difficult?
Invasive - would need to isolate DNA, then sequence the
DNA to see if the DNA sequence has changed compared
to DNA found in the non targeted somatic cells.
8. Explain why some physicians and clinicians feel that gene therapy is a distant future
consideration.
There have been many failures along with the successful cases. Many
are still violently opposed to the experimentation.
As of 2007, there have been 1,340 completed or approved gene
therapy clinical trials in 28 different countries worldwide. The United
States is responsible for 64.2% of all gene therapy trials. Other major
contributors include the United Kingdom at 11.1%, Germany at 5.5%,
and Switzerland at 3.1% (Edelstein et al. 2007).
Exit Slip
 Have
you already started studying
for your test? Why or why not?
Homework
Do Now/Exit Slip Reflection
 Parent Signature
 EC Review Guide


Unit 2 Test on Tuesday
Video
 https://www.youtube.com/watch?v=
jAhjPd4uNFY