teacher guide: vector selector -...
TRANSCRIPT
Genetic Science Learning Center, 15 North 2030 East, Salt Lake City, UT 84112 © 2002 University of Utah
http://gslc.genetics.utah.edu
ACTIVITY OVERVIEW
Teacher Guide: Vector Selector
Abstract:
Acting as employees of a vector supply
company, students research a disease
that is a candidate for gene therapy and
determine the best vector to use. They
must then present their choice and the
reasoning behind it in order to help
“sell” the vector to a biotech company
developing a therapy for this disease.
Module:
Gene Therapy: Molecular Bandage?
Prior Knowledge Needed:
DNA structure and function; mutation;
gene expression; basic cell structure and
function; viral ligands bind to receptors of
specifi c cell types; ex vivo; in vivo
Key Concepts:
Vectors; in vivo; ex vivo; cell targeting;
immune response
Materials:
Student Pages, computers and/or library
for research (optional), media equipment as
necessary for student presentations,
art supplies as needed for student
presentations
Appropriate For:
Ages: 15 - 20
USA grades: 10 - 14
Prep Time:
30 minutes to review gene therapy scenarios
and copy
Class Time:
2-4 class periods depending on student
research and presentations
Activity Overview Web Address:
http://gslc.genetics.utah.edu/teachers/tindex/
overview.cfm?id=vselector
Other activities in the Gene Therapy: Molecular Bandage? module can be
found at:
http://gslc.genetics.utah.edu/teachers/tindex/
Genetic Science Learning Center, 15 North 2030 East, Salt Lake City, UT 84112 © 2002 University of Utah
http://gslc.genetics.utah.edu
Teacher Guide: Vector Selector
Pedagogy
A. Learning Objectives
B. Background Information
C. Teaching Strategies
Page
1-3
Additional Resources
A. Activity Resources
4
Materials
A. Detailed Materials List
4
Standards
A. U.S. National Science Education Standards
B. AAAS Benchmarks for Science Literacy
C. Utah Secondary Science Core Curriculum
4-5
Teacher References
A. Vector Choices Answer Key
6
Student Pages
• Vector Types Table
• Situation/Assignment One
• Situation/Assignment Two
• Situation/Assignment Three
• Situation/Assignment Four
• Situation/Assignment Five
• Assessment Rubric
• Blank Vector Types Table
S-1
S-2
S-3
S-4
S-5
S-6
S-7
S-8
TABLE OF CONTENTS
http://gslc.genetics.utah.edu
Genetic Science Learning Center, 15 North 2030 East, Salt Lake City, UT 84112 © 2002 University of Utah
Teacher Guide: Vector Selector
1 of 6
I. PEDAGOGY
A. Learning Objectives
• Students will design a solution to a problem by applying their knowledge of
gene therapy and vectors.
• Students will think critically about the properties of various vectors.
• Students will communicate their thought process and rationale in choosing a
specifi c vector for a given gene therapy scenario.
B. Background Information
Activity Information
This activity is designed for use after students have studied the different types of
vectors used in gene therapy and the advantages/disadvantages of each type.
Students also need a basic understanding of gene therapy delivery techniques
(ex vivo versus in vivo), and the genetic disorder presented in their assigned
scenario. The “Vector Types” table (page S-1), and the online portion of the
Gene Therapy: Molecular Bandage? module on the Genetic Science Learning
Center website (http://gslc.genetics.utah.edu) will provide most of the necessary
background information. Basic information about the specifi c genetic disorder is
provided in the scenarios. Also see Additional Resources in the online portion of
the Gene Therapy: Molecular Bandage? module for more information on specifi c
disorders.
There is a lot of fl exibility in the type of presentation students may be assigned
to give depending on available time, technology and desired complexity of
presentation. Students may also work individually or in groups. Some ideas
include:
• Group discussions, then oral reports about what vector they would choose
• Oral “sales pitch” including visuals
• Poster or brochure to market their vector
• Written prospectus describing the vector and its use in gene therapy
• Multi-media presentation
A rubric for presentations (page S-7) and a vector choices answer key (page 6)
is provided for your convenience.
Five different gene therapy situations are included in this activity. You may wish
to assign different situations to each group of students, or assign the same
situation to all groups.
http://gslc.genetics.utah.edu
Genetic Science Learning Center, 15 North 2030 East, Salt Lake City, UT 84112 © 2002 University of Utah
Teacher Guide: Vector Selector
2 of 6
Scientifi c Information
Scientists refer to DNA delivery vehicles as vectors. Each vector is designed
to target specifi c cells. Traditionally, vectors have been derived from viruses,
including retroviruses, adenoviruses, adeno-associated viruses, and herpes
simplex viruses. Components of the virus that cause disease are removed and
the gene the researcher wants to be delivered is inserted. The transfer of DNA
into a cell by a modifi ed infectious virus is called transfection. This distinguishes
it from infection, by which a virus inserts its own DNA or RNA into a cell. Non-
viral gene delivery approaches being examined include the use of liposomes,
(lipid-based pockets that can carry plasmid DNA) or simply naked DNA with no
carrier. Each vector and method of delivery varies depending on the specifi c
disorder the therapy aims to treat. Three important factors to consider when
choosing a vector for gene therapy are:
1. Will the gene fi t into the vector?
2. Will the vector target the right type of tissue?
3. Does the vector work within the appropriate cell cycle stage to
affect the disease?
There are two basic ways to deliver a gene into a group of cells in a patient’s
body. The fi rst is to inject the vector into the body and specifi cally target those
cells. This is called an in vivo approach.
The second way, called ex vivo, is to deliver the gene to cells while they are
outside the body using the following procedure:
1. Isolate the desired cells from the body.
2. Culture the cells in a Petri dish, in the laboratory.
3. Deliver the gene to the cells (using an appropriate vector),
activate it, and make sure it integrates properly into the cells.
4. Put the genetically modifi ed cells back into the proper place in the
body, ensure that they survive there, and let them get to work.
Each of the vectors and methods has its strengths and limitations for gene
delivery. Success in gene therapy depends on the effi cient delivery of the
correct gene to the correct cells in the correct tissue. Thus, the selection of a
particular vector is highly dependent upon its properties and the characteristics
of the disease undergoing treatment. See Teacher Reference (page 6) for
information about how each vector type would or would not work for each
situation included in this activity.
http://gslc.genetics.utah.edu
Genetic Science Learning Center, 15 North 2030 East, Salt Lake City, UT 84112 © 2002 University of Utah
Teacher Guide: Vector Selector
3 of 6
C. Teaching Strategies
1. Timeline
• One day before activity:
- Decide what type of presentation to assign (see suggestions in
Background, page 1)
- Decide which situation or situations to use
- Copy situations (Student Pages S-2 through S-7), one per student or
group
- Copy the Assessment Rubric (page S-7) and Vector Types Table (page
S-1), make one copy per student (optional)
• Day of activity:
- Divide class into groups
- Hand out copies of situations (Pages S-2 through S-6)
- Provide time for students to complete the task and prepare a
presentation
2. Classroom Implementation
• Introduce the overall assignment background to the class (located at the top of
each situation).
• Divide the class into groups.
• Assign each group a specifi c gene therapy situation.
• Explain the presentation requirements.
◦ If using it, hand out the presentation assessment rubric (Page S-7).
• Hand out copies of the Vector Types Table (page S-1).
• Provide time for groups to research and prepare their presentations.
• Provide time for groups to present.
3. Extensions
• Have students research gene therapies currently being studied and their
relative success.
4. Adaptations
• You may want to have students fi ll out the blank Vector Types table (S-
8) on their own. This information is located online in the Vector Toolbox
found in the “Tools of the Trade” section of the Gene Therapy: Molecular
Bandage? module on the Genetic Science Learning Center website (http:
//gslc.genetics.utah.edu). You may project the Vector Toolbox to the whole
class or bring your class to the computer lab to visit this section individually.
http://gslc.genetics.utah.edu
Genetic Science Learning Center, 15 North 2030 East, Salt Lake City, UT 84112 © 2002 University of Utah
Teacher Guide: Vector Selector
4 of 6
• Use this activity as a prompt for students to research basic information about
gene therapy on their own.
• Have students carry out this activity individually, rather than in groups.
5. Assessment Suggestions
• Use the presentations as an assessment. (See page S-7 for rubric)
• Because this activity requires students to use what they know about gene
therapy, it may be used as an overall assessment for a gene therapy unit.
6. Common Misconceptions
• Students sometimes think that gene therapy replaces a faulty gene with a
functional copy. It is important that they understand this is not the case. Gene
therapy endeavors to add a functional copy of the gene in the hope that its
function will counteract the effect of the faulty gene.
II. ADDITIONAL RESOURCES
A. Activity Resources linked from the online Activity Overview at:
http://gslc.genetics.utah.edu/teachers/tindex/overview.cfm?id=vselector
• Website: Gene Therapy: Molecular Bandage? - information about the idea
behind gene therapy, techniques, current status of trials and other related
issues.
• Website: Gene Therapy: Molecular Bandage? - Additional Resources -
information about the specifi c genetic disorders addressed in this activity’s
scenarios.
• Website: Genetic Disorder Corner: What is NF 1? - information about
Neurofi bromatosis 1.
III. MATERIALS
A. Detailed Materials List
• Student Pages S-1 through S-6, one per student or groups
• Assessment rubric, page S-7 (optional)
• Computers (optional) if providing time for online research
• Art supplies (optional) as necessary for presentations
• Media equipment (optional) as necessary for presentations
http://gslc.genetics.utah.edu
Genetic Science Learning Center, 15 North 2030 East, Salt Lake City, UT 84112 © 2002 University of Utah
Teacher Guide: Vector Selector
5 of 6
IV. STANDARDS
A. U.S. National Science Education Standards
Grades 9-12:
• Content Standard C: Life Science - The Molecular Basis of Heredity; in all
organisms, the instructions for specifying the characteristics of the
organism are carried in DNA.
B. AAAS Benchmarks for Science Literacy
Grades 9-12:
• The Living Environment: Heredity - genes are segments of DNA molecules;
inserting, deleting, or substituting DNA segments can alter genes; an altered
gene may be passed on to every cell that develops from it; the resulting
features may help, harm, or have little or no effect on the offspring’s success in
its environment.
• The Human Organism: Physical Health - faulty genes can cause body parts or
systems to work poorly.
• The Designed World: Health Technology - knowledge of genetics is opening
whole new fi elds of health care.
C. Utah Secondary Science Core Curriculum
Intended Learning Outcomes for Ninth to Twelfth Grade Science
Students will:
4. Demonstrate Awareness of the Social and Historical Aspects of Science
a. Provide relevant data to support their inferences and conclusions
Biology (9-12)
Standard 4: Students will understand that genetic information coded in DNA is
passed from parents to offspring by sexual and asexual reproduction. The basic
structure of DNA is the same in all living things. Changes in DNA may alter ge-
netic expression.
Objective 3: Explain how the structure and replication of DNA are essential to
heredity and protein synthesis.
- Research, report, and debate genetic technologies that may improve the
quality of life (e.g., genetic engineering, cloning, gene splicing).
http://gslc.genetics.utah.edu
Genetic Science Learning Center, 15 North 2030 East, Salt Lake City, UT 84112 © 2002 University of Utah
Teacher Guide: Vector Selector
6 of 6
V. CREDITS
Activity created by:
Jennifer Burbank, Weber High School, Ogden, Utah
Steve Scheidell, Murray High School, Murray, Utah
Molly Malone, Genetic Science Learning Center
Mel Limson, Genetic Science Learning Center
Harmony Starr, Genetic Science Learning Center (illustrations)Steven Kiger, Genetic Science Learning Center (illustrations)
Funding:
Funding for this module was provided by a Science Education Partnership Award
(No. 1 R25 RR16291) from the National Center for Research Resources, a
component of the National Institutes of Health.
http://gslc.genetics.utah.edu
Genetic Science Learning Center, 15 North 2030 East, Salt Lake City, UT 84112 © 2002 University of Utah
Teacher Reference: Vector Choices Answer Key
7 of 6
RE
TR
OV
IRU
SA
DE
NO
VIR
US
AD
EN
O-
AS
SO
CIA
TE
D
VIR
US
HE
RP
ES
SIM
PL
EX
VIR
US
LIP
OS
OM
EN
AK
ED
DN
A
Sit
uati
on
On
e
N
FS
Co
.
NO
T P
OS
SIB
LE
The N
F1 g
ene is e
n-
co
de
d b
y 8
,45
4 b
ase
pairs. T
he g
ene is
too larg
e to fi t in this
type o
f vecto
r.
NO
T P
OS
SIB
LE
The N
F1 g
ene is e
n-
co
de
d b
y 8
,45
4 b
ase
pairs. T
he g
ene is
too larg
e to fi t in this
type o
f vecto
r.
NO
T P
OS
SIB
LE
T
he N
F1 g
ene is e
n-
co
de
d b
y 8
,45
4 b
ase
pairs. T
he g
ene is
too larg
e to fi t in this
type o
f vecto
r.
NO
T P
OS
SIB
LE
Although the N
F1
gene c
ontr
ibute
s t
o
part
s o
f th
e n
erv
ous
syste
m, th
e H
SV
will
not in
tegra
te into
the
host cell’
s g
enom
e.
An
im
mu
ne
re
sp
on
se
is p
ossib
le.
NO
T P
OS
SIB
LE
Ve
cto
r has n
o c
ell
targ
et specifi c
ity
NO
T P
OS
SIB
LE
The N
F1 g
ene is e
n-
coded b
y 8
,454 b
ase
pairs. T
he g
ene is
too larg
e to fi t in this
type o
f vecto
r.
Sit
uati
on
Tw
o
CellM
art
PO
SS
IBL
E
The v
ecto
r can b
e
intr
od
uce
d in
to b
loo
d
ste
m c
ells
, w
hic
h
div
ide
fre
qu
en
tly.
How
ever, the g
ene
may inte
gra
te into
the
ho
st ce
ll’s g
en
om
e in
ran
do
m lo
ca
tio
ns a
nd
dis
rup
t a
no
the
r g
en
e.
PO
SS
IBL
ET
he v
ecto
r can b
e
intr
od
uce
d in
to b
loo
d
ste
m c
ells
, w
hic
h d
i-
vid
e fre
quently.
H
ow
-
ever, it m
ight cause
an
im
mu
ne
re
sp
on
se
.
NO
T P
OS
SIB
LE
Th
e b
loo
d-c
lott
ing
ge
ne
is e
nco
de
d b
y
7,3
50
ba
se
pa
irs.
The g
ene is too larg
e
to fi t in this
type o
f
ve
cto
r.
NO
T P
OS
SIB
LE
Blo
od c
lottin
g fac-
tors
are
not part
of
the n
erv
ous s
yste
m.
They a
re p
art
of th
e
circula
tory
syste
m.
NO
T P
OS
SIB
LE
Ve
cto
r has n
o c
ell
targ
et specifi c
ity
NO
T P
OS
SIB
LE
Ve
cto
r has n
o c
ell
targ
et specifi c
ity.
Sit
uati
on
Th
ree
Om
a, In
c.
NO
T P
OS
SIB
LE
Ve
cto
r in
fects
only
div
idin
g c
ells
. L
un
g
cells
div
ide s
low
ly, if
at a
ll.
PO
SS
IBL
ET
he g
enes o
f th
is
vecto
r do n
ot in
-
tegra
te into
the
ge
no
me
. R
ep
ea
ted
treatm
ents
would
be
necessary
. A
n im
-
mu
ne
re
sp
on
se
is
po
ssib
le.
PO
SS
IBL
EV
ecto
r is
effective a
t
en
terin
g lu
ng
ce
lls,
an
d typ
ica
lly d
oe
s
not cause a
n im
mune
resp
on
se
.
NO
T P
OS
SIB
LE
Lu
ng
ce
lls a
re n
ot
part
of th
e n
erv
ous
syste
m. T
hey a
re
part
of th
e r
espirato
ry
syste
m
NO
T P
OS
SIB
LE
Ve
cto
r has n
o c
ell
targ
et specifi c
ity
NO
T P
OS
SIB
LE
Ve
cto
r has n
o c
ell
targ
et specifi c
ity.
Sit
uati
on
Fo
ur
SB
G, In
c.
PO
SS
IBL
E
The v
irus infe
cts
div
idin
g c
ells
, b
ut
in-
tegra
tes into
the h
ost
ce
ll’s g
en
om
e in
ra
n-
do
m lo
ca
tio
ns, w
hic
h
mig
ht dis
rupt anoth
er
ge
ne
. A
n im
mu
ne
re
-
sp
on
se
is p
ossib
le.
PO
SS
IBL
ET
he v
irus d
oes n
ot
inte
gra
te into
the
ge
no
me
, a
nd
so
,
rep
ea
ted
tre
atm
en
ts
would
be n
ecessary
.
Als
o, m
ay c
ause a
n
imm
un
e r
esp
on
se
.
PO
SS
IBL
EV
ecto
r in
fects
cells
effi c
ien
tly,
usu
ally
inte
gra
tes in
to a
sp
e-
cifi c
, non-d
isru
ptive
loca
tio
n, a
nd
typ
ica
lly
do
es n
ot ca
use
an
imm
un
e r
esp
on
se
.
NO
T P
OS
SIB
LE
T-c
ells
are
not part
of
the n
erv
ous s
yste
m.
They a
re p
art
of th
e
imm
une s
yste
m.
NO
T P
OS
SIB
LE
Ve
cto
r has n
o c
ell
tar-
get spe
cifi c
ity.
Ente
rs
cells
le
ss e
ffectively
than v
iruses.
NO
T P
OS
SIB
LE
Ve
cto
r has n
o c
ell
tar-
get specifi c
ity.
Ente
rs
cells
less e
ffectively
tha
n v
iru
se
s.
Sit
uati
on
Fiv
e
CF, In
c.
NO
T P
OS
SIB
LE
Ve
cto
r in
fects
only
div
idin
g c
ells
. L
un
g
cells
div
ide s
low
ly, if
at all
PO
SS
IBL
ET
he v
ecto
r is
effective
at
en
terin
g n
on
-div
id-
ing
ce
lls lik
e lu
ng
ce
lls, b
ut ca
n c
au
se
an
im
mu
ne
re
sp
on
se
.
PO
SS
IBL
ET
he v
ecto
r is
effective
at
en
terin
g n
on
-div
id-
ing
ce
lls lik
e lu
ng
ce
lls, a
nd
typ
ica
lly
do
es n
ot ca
use
an
imm
un
e r
esp
on
se
.
NO
T P
OS
SIB
LE
Lu
ng
ce
lls a
re n
ot
part
of th
e n
erv
ous
syste
m. T
hey a
re
part
of th
e r
espirato
ry
syste
m.
NO
T P
OS
SIB
LE
Ve
cto
r has n
o c
ell
tar-
get spe
cifi c
ity.
E
nte
rs
cells
le
ss e
ffectively
than v
iruses.
NO
T P
OS
SIB
LE
Ve
cto
r has n
o c
ell
tar-
get specifi c
ity.
Ente
rs
cells
less e
ffectively
tha
n v
iru
se
s.
http://gslc.genetics.utah.edu© 2003 University of Utah
Name Date
Permission granted for classroom use. S-1
RE
TR
OV
IRU
SA
DE
NO
VIR
US
AD
EN
O-A
SS
OC
IAT
ED
VIR
US
HE
RP
ES
SIM
PL
EX
VIR
US
LIP
OS
OM
EN
AK
ED
DN
A
Ho
w t
he v
ecto
r
ca
rrie
s g
en
eti
c
ma
teri
al
In the form
of R
NA
rath
er
than D
NA
.
The m
ost fa
mous is
Hum
an Im
munodefi cie
ncy
Virus (
HIV
), w
hic
h c
auses
AID
S.
Double
-str
anded D
NA
. The c
om
mon c
old
virus.
Sin
gle
-str
anded D
NA
Sin
gle
-str
anded D
NA
Pla
sm
id D
NA
packaged into
min
iatu
re lip
id
–based p
ockets
that can fuse to
the c
ell’
s o
wn
mem
bra
nes,
The D
NA
is
rele
ased a
nd
transport
ed into
the n
ucle
us
Pla
sm
id D
NA
mole
-
cule
all
by its
elf that
can b
e taken u
p
by s
om
e c
ells
, and
transport
ed into
the
nucle
us
Maxim
um
len
gth
of
DN
A t
hat
can
be
insert
ed
in
to v
ecto
r
8, 000 b
ase p
airs
7,5
00 b
ase p
airs
5,0
00 b
ase p
airs
20,0
00 b
ase p
airs
AD
VA
NTA
GE
S
+ Infe
cts
only
div
idin
g c
ells
+ Infe
cts
both
div
idin
g
and n
on-d
ivid
ing c
ells
very
effectively
+ P
ossib
le to targ
et
specifi c
cell
types b
y
engin
eering p
rote
ins
on the v
irus s
urf
ace
to r
ecogniz
e s
pecia
l
pro
tein
s o
n the targ
et
cell’
s s
urf
ace
+ D
oes n
ot cause illn
ess in h
um
ans
+ Infe
cts
a w
ide r
ange o
f div
idin
g a
nd n
on-
div
idin
g c
ell
types v
ery
effectively
+ N
eed the a
ssis
tance o
f a “
help
er”
virus to
replic
ate
them
selv
es insid
e c
ells
+ P
ossib
le to targ
et specifi c
cell
types b
y
engin
eering p
rote
ins o
n the v
irus s
urf
ace
to r
ecogniz
e s
pecia
l pro
tein
s o
n the targ
et
cell’
s s
urf
ace
+ Inte
gra
tes into
the h
ost cell’
s g
enom
e;
95%
of th
e tim
e, it w
ill inte
gra
te into
a
specifi c
regio
n o
n C
hro
mosom
e 1
9, gre
atly
reducin
g the c
hance that in
tegra
tion w
ill
dis
rupt th
e function o
f oth
er
genes in the
cell
+ T
ypic
ally
will
not cause a
n im
mune
response
+ Infe
cts
cells
of th
e
nerv
ous s
yste
m
+ W
ill n
ot in
tegra
te
into
the h
ost cell’
s
genom
e, but is
a
circula
r pie
ce o
f D
NA
that re
plic
ate
s w
hen
the c
ell
div
ides; w
ill
sta
y in the c
ell’
s
nucle
us for
a long
tim
e
+ W
ill n
ot dis
rupt
the function o
f oth
er
genes in the h
ost cell
+ W
ill n
ot
genera
te
an im
mune
response
+ B
etter
suited
for
ex v
ivo
gene thera
py
appro
aches
+ W
ill n
ot genera
te
an im
mune
response
+ G
enera
lly n
ot
toxic
DIS
AD
VA
NTA
GE
S
- In
tegra
tes into
the h
ost cell’
s
genom
e in r
andom
locations;
mig
ht in
tegra
te
into
a p
lace
where
it dis
rupts
anoth
er
gene -
Can
cause a
n im
mune
response
- W
ill n
ot in
tegra
te
into
the h
ost cell’
s
genom
e; th
e c
ell
will
dis
card
the v
irus a
nd
gene a
ctivation w
ill
be lost
- C
an c
ause a
n
imm
une r
esponse
- 5%
chance o
f in
tegra
ting a
nd d
isru
pting
the function o
f oth
er
genes in the c
ell
- C
an c
ause a
n
imm
une r
esponse in
the p
atient
- N
ot specifi c
for
any c
ell
type
- E
nte
r, c
ells
far
less e
ffectively
than v
iruses
- W
ill n
ot
inte
gra
te into
the h
ost cell’
s
genom
e
- N
ot specifi c
for
any c
ell
type
- E
nte
r, c
ells
far
less e
ffectively
than
viruses
- W
ill n
ot in
tegra
te
into
the h
ost cell’
s
genom
e
- U
nsta
ble
in m
ost
body tis
sues
Vecto
r T
yp
es
http://gslc.genetics.utah.edu© 2003 University of Utah
Name Date
Permission granted for classroom use. S-2
The ABDS marketing department has identifi ed NFS Co. as a biotech company that is developing gene therapies.
NFS Co. is focusing on neurofi bromatosis type 1 which is caused by a mutation in the NF1 gene (8,454 base
pairs) on chromosome 17. This disorder causes tumors to grow on nerves, skin changes and bone deformities.
NFS Co. is looking for a vector that could target non-dividing nerve cells and insert a transgene stably into the
chromosome.
Prepare a sales pitch to suggest a vector.
You are a researcher at Advanced Bio Delivery Systems (ABDS), a
company that supplies vectors for use in gene therapy. The marketing
department has come to you for assistance. They would like to sell
the vectors made by ABDS to biotech companies pursuing specifi c
gene therapies. It is your job to research the disorders these biotech
companies are targeting and decide on a vector that has potential to work. You will be presenting your
fi ndings to the marketing department for approval. There may be more than one vector possibility, so
your job is to choose what you feel is the best one and defend your position. Your company stands to
make a considerable profi t if you are able to “sell” their vector for use in research.
Some things to consider:
• What are important characteristics of the disease being investigated (genetic defect, body
system affected…)?
• Will the gene fi t into the vector?
• What types of cells would the therapy need to target (dividing/non-dividing)?
• What will happen to the genetic material once it is inserted into the cells?
• Does gene expression need to last a long time?
• What kinds of safety issues need to be considered before recommending a vector type?
http://gslc.genetics.utah.edu© 2003 University of Utah
Name Date
Permission granted for classroom use. S-3
The ABDS marketing department has identifi ed CellMart as a biotech company that is developing gene therapies.
CellMart is searching for a vector to treat hemophilia, a bleeding disorder caused by a defi ciency in one of the
blood clotting factors. This results in excessive bleeding. CellMart intends to isolate blood stem cells, which
divide frequently, and introduce the vector ex vivo. The gene they plan to use is 7,350 base pairs long.
Prepare a sales pitch to suggest a vector.
You are a researcher at Advanced Bio Delivery Systems (ABDS), a
company that supplies vectors for use in gene therapy. The marketing
department has come to you for assistance. They would like to sell
the vectors made by ABDS to biotech companies pursuing specifi c
gene therapies. It is your job to research the disorders these biotech
companies are targeting and decide on a vector that has potential to work. You will be presenting your
fi ndings to the marketing department for approval. There may be more than one vector possibility, so
your job is to choose what you feel is the best one and defend your position. Your company stands to
make a considerable profi t if you are able to “sell” their vector for use in research.
Some things to consider:
• What are important characteristics of the disease being investigated (genetic defect, body
system affected…)?
• Will the gene fi t into the vector?
• What types of cells would the therapy need to target (dividing/non-dividing)?
• What will happen to the genetic material once it is inserted into the cells?
• Does gene expression need to last a long time?
• What kinds of safety issues need to be considered before recommending a vector type?
CellMart
http://gslc.genetics.utah.edu© 2003 University of Utah
Name Date
Permission granted for classroom use. S-4
The ABDS marketing department has identifi ed Oma, Inc. as a biotech company that is developing gene therapies.
Oma Inc. would like to target non-small cell lung cancer (75% of all lung cancers) with gene therapy. They need a
vector to introduce p53, a tumor suppressor gene (2,150 base pairs long), directly into the tumor. The vector must
target the lung cells directly and the potential immune response must be considered.
Prepare a sales pitch to suggest a vector.
You are a researcher at Advanced Bio Delivery Systems (ABDS), a
company that supplies vectors for use in gene therapy. The marketing
department has come to you for assistance. They would like to sell
the vectors made by ABDS to biotech companies pursuing specifi c
gene therapies. It is your job to research the disorders these biotech
companies are targeting and decide on a vector that has potential to work. You will be presenting your
fi ndings to the marketing department for approval. There may be more than one vector possibility, so
your job is to choose what you feel is the best one and defend your position. Your company stands to
make a considerable profi t if you are able to “sell” their vector for use in research.
Some things to consider:
• What are important characteristics of the disease being investigated (genetic defect, body
system affected…)?
• Will the gene fi t into the vector?
• What types of cells would the therapy need to target (dividing/non-dividing)?
• What will happen to the genetic material once it is inserted into the cells?
• Does gene expression need to last a long time?
• What kinds of safety issues need to be considered before recommending a vector type?
Omainc.
http://gslc.genetics.utah.edu© 2003 University of Utah
Name Date
Permission granted for classroom use. S-5
The ABDS marketing department has identifi ed SCID-BE-GONE (SBG), Inc. as a biotech company that is
developing gene therapies. SBG is interested in developing a delivery system that will insert a good ADA gene
(1,498 base pairs long) in T-cells of patients who have SCID (severe combined immune defi ciency). SBG plans
to harvest T-cells from a patient and use an ex-vivo technique to insert a functional ADA gene. As the transfected
cells begin to produce the ADA protein, they will be retuned to the patient. The company is having trouble fi nding
a vector to deliver their gene.
Prepare a sales pitch to suggest a vector.
You are a researcher at Advanced Bio Delivery Systems (ABDS), a
company that supplies vectors for use in gene therapy. The marketing
department has come to you for assistance. They would like to sell
the vectors made by ABDS to biotech companies pursuing specifi c
gene therapies. It is your job to research the disorders these biotech
companies are targeting and decide on a vector that has potential to work. You will be presenting your
fi ndings to the marketing department for approval. There may be more than one vector possibility, so
your job is to choose what you feel is the best one and defend your position. Your company stands to
make a considerable profi t if you are able to “sell” their vector for use in research.
Some things to consider:
• What are important characteristics of the disease being investigated (genetic defect, body
system affected…)?
• Will the gene fi t into the vector?
• What types of cells would the therapy need to target (dividing/non-dividing)?
• What will happen to the genetic material once it is inserted into the cells?
• Does gene expression need to last a long time?
• What kinds of safety issues need to be considered before recommending a vector type?
http://gslc.genetics.utah.edu© 2003 University of Utah
Name Date
Permission granted for classroom use. S-6
The ABDS marketing department has identifi ed CF, Inc. (CFI) as a biotech company that is developing gene
therapies. CFI is interested in developing a delivery system that will insert a good CFTR gene in the lung cells
of patients who have cystic fi brosis. The CFTR gene (4,443 base pairs long) codes for the protein that controls
ion transfer across cell membranes. CFI is developing an in vivo system to deliver the functional gene copy.
Challenges that face CFI are getting the vector to the lung cells and dealing with the immune response mounted
as the vector enters the lungs.
Prepare a sales pitch to suggest a vector.
You are a researcher at Advanced Bio Delivery Systems (ABDS), a
company that supplies vectors for use in gene therapy. The marketing
department has come to you for assistance. They would like to sell
the vectors made by ABDS to biotech companies pursuing specifi c
gene therapies. It is your job to research the disorders these biotech
companies are targeting and decide on a vector that has potential to work. You will be presenting your
fi ndings to the marketing department for approval. There may be more than one vector possibility, so
your job is to choose what you feel is the best one and defend your position. Your company stands to
make a considerable profi t if you are able to “sell” their vector for use in research.
Some things to consider:
• What are important characteristics of the disease being investigated (genetic defect, body
system affected…)?
• Will the gene fi t into the vector?
• What types of cells would the therapy need to target (dividing/non-dividing)?
• What will happen to the genetic material once it is inserted into the cells?
• Does gene expression need to last a long time?
• What kinds of safety issues need to be considered before recommending a vector type?
INC.
http://gslc.genetics.utah.edu© 2003 University of Utah
Name Date
Permission granted for classroom use. S-7
Vector Selector: Assessment Rubric
CATEGORY 4 3 2 1
Amount of
information on
disease
Important
characteristics
of the disease
are addressed
and described in
detail.
Most of the
important
characteristics of
the disease are
addressed and
described.
Important
characteristics
of the disease
are addressed
and described
marginally.
Only one
characteristic
of the disease
is addressed.
Amount of
information on
vector
Important
characteristics
of the vector
are addressed
and described in
detail.
Most of the
important
characteristics
of the vector are
addressed and
described.
Important
characteristics
of the vector
are addressed
and described
marginally.
Only one
characteristic
of the vector is
addressed.
Targeting
issues
Vector chosen
will bind to the
target cells.
Description
of targeting
strategy is
detailed.
Vector chosen
will bind to the
target cells.
Description
of targeting
strategy is
somewhat
vague.
Vector chosen
will bind to the
target cells.
Description of
targeting strategy
is incomplete.
Vector chosen
will not bind to
the target cells.
Integration of
genetic material
Includes detailed
description of
what will happen
to the DNA.
Description of
what will happen
to the DNA is
correct, but
described in less
detail
Description of
what will happen
to the DNA is
lacking some
information.
Description
of what will
happen to the
DNA is unclear
or incorrect.
Vector Choice
Provides ideas
that support
choice of vector.
Provides ideas
that support
choice of vector.
Provides
one idea that
supports choice
of vector.
No ideas
presented that
support choice
of vector.
http://gslc.genetics.utah.edu© 2003 University of Utah
Name Date
Permission granted for classroom use. S-8
RE
TR
OV
IRU
SA
DE
NO
VIR
US
AD
EN
O-A
SS
OC
IAT
ED
VIR
US
HE
RP
ES
SIM
PL
EX
VIR
US
LIP
OS
OM
EN
AK
ED
DN
A
Ho
w t
he v
ecto
r
ca
rrie
s g
en
eti
c
ma
teri
al
Maxim
um
len
gth
of
DN
A t
hat
can
be
insert
ed
in
to v
ecto
r
AD
VA
NTA
GE
S
DIS
AD
VA
NTA
GE
S
Vecto
r T
yp
es