Gene Therapy

butterbeanscubaBiotechnologie

12 déc. 2012 (il y a 8 années et 7 mois)

471 vue(s)

Genetic Engineering of
Bacteria to Produce
Human Proteins

human

cell

Human insulin
gene

bacterial DNA

Insert
bacterial/human DNA
into bacterial cell

Bacteria produces
human
INSULIN
protein
, which is
purified and given
to patients

Genetic Engineering of
Bacteria to Produce
Human Proteins

human cell

Human
blood clotting gene

bacterial DNA

Insert
bacterial/human DNA
into bacterial cell

Bacteria produces
BLOOD
CLOTTING
PROTEIN
,

which is purified
and given to
patients

Genetic Engineering of
Bacteria to Produce
Human Proteins

human cell

bacterial DNA

Insert
bacterial/human DNA
into bacterial cell

Human
erythropoietin gene

Bacteria produces
ERYTHROPOIETIN
PROTEIN
,

which is purified and
given to patients


An experimental treatment
which attempts to treat a disease
by inserting a gene into the cells
of a human



>3000 patients have been
treated in >300 clinical trials
around the globe



No gene therapy treatment is yet
approved for general use

Gene Therapy

Jesse Gelsinger


-

received gene therapy for OTC
Deficiency

-

died September 1999

Gene Therapy for OTC
Deficiency

human

cell

Normal
OTC gene

Insert the OTC
gene into a virus

Deliver the virus directly
into vein leading to liver


virus infects liver
cells and delivers gene


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灥牳潮

S
evere
C
ombined
I
mmuno
d
eficiency

(SCID)



The first disease to be
treated with gene therapy



Persons with SCID lack a
functional immune system
and are susceptible to severe
infections

David Vetter

(1972
-
1984)


-

lived in a sterile “plastic
bubble” because had a
single defective gene that
caused Severe Combined
Immunodeficiency (SCID)

Gene Therapy for SCID

Remove blood
cells from
patient

Infect blood
cells with a
virus that has
been genetically
engineered to
carry the
corrected SCID
gene

Re
-
infuse the
blood cells
into the patient

Ashanthi DeSilva

-

inherited SCID


-
received 1
st

ever gene
therapy in 1990

Gene Therapy for SCID

Using Long
-
lived
Umbilical Cord Blood
Cells

Infect cord
blood cells with
a virus that has
been genetically
engineered to
carry the
corrected SCID
gene

Remove blood
cells from the
umbilical cord

Re
-
infuse the
cord blood cells
into the patient

Andrew Gobea

(& mom)

-

inherited SCID

-

received 1
st

ever gene
therapy using long
-
lived cord
blood cells

Other Partially
Successful Gene
Therapy Trials



therapy to induce new blood
vessel growth in patients with
blocked heart arteries or with
other circulatory problems



therapy to induce brain tumor
cells to “commit suicide”

Ethical Questions
Concerning Gene
Therapy


Should treatments be used to change
the appearance, intelligence, or
athletic ability of healthy people?



Should parents be allowed to
genetically manipulate their children
before birth?



Should gene therapy be allowed that
alters germ
-
line cells (reproductive
cells that will pass the altered genes
to future generations)?