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Transcript
Consider:
 We get all of our traits from all the generations
before us.
 Naturally, we each have our own probability of
something in our body not working as it should
the second we are born.
 These include diseases we can get, to small things
like poor sight.
 For millennia, we have not been able to change the
base chances of us catching a disease, we could
only try our best to help prevent it.
 There are two cases in which Gene Therapy
would be used.
 A requirement is, however, that there must be
some disease or defect that needs to be fixed.
 Either the person is completely missing the
needed gene, or the current gene they have is not
working correctly.
 Nevertheless, the procedure generally stays the
same, unless all that is needed is to “kick out” the
bad genes.
Say we are genetically modifying someone’s bone
marrow to have more white blood cells.
I.
Scientists take out the defective genes, and modify them into
healthy genes.
II. Scientists start by attaching the normal, healthy gene they
want to give the patient to a harmless virus called a vector.
III. They remove genetic material from the vector and replace it
with genetic instructions to make a healthy copy of the
person’s missing gene.
IV. They then mix the vector and the new gene with bone marrow
or blood from the patient.
V. The vector carrying the normal gene will penetrate the stem
cells in the patient’s bone marrow/blood and replace the
defective gene with the healthy gene.
VI. Doctors then grow these corrected cells in an incubator (this
step is basically spreading the healthy genes). Once they have
enough, they inject them into the patient. The bone marrow will
gradually absorb them. The new cells can then start making
healthy white blood cells able to fight infection.
By: Molly
By: Bharadwaj
Sudarsan
Weiman
 Gene therapy uses genes to treat diseases.
 Previously, surgery, drugs, and other sorts of
treatments were given to treat diseases.
 Gene Therapy involves injecting genes into the
patient so that the genes can treat or prevent the
disease(s) (like cancer, diabetes, etc.).
 They can replace the mutated gene with a healthy
version of it, “knock out” an improperly
functioning mutant gene, or introducing a new
gene to help fight the mutant gene.
 They also normally have no noticeable side effects.
 Previously, in cancer, the only 3 forms of treatment were
surgery (surgically removing tumors), radiation therapy
(Using X-rays to destroy tumors), and chemotherapy.
 Now, with gene therapy, one can just prevent cancer by
injecting him/herself with a healthy gene. This procedure
can be done to many other diseases like aneurism, thyroid,
diabetes, etc.
 There used to be no cure for Chronic Granulomatous
Disorder, a genetic disease in the immune system that
makes patients unable to fight off bacterial and fungal
infections.
 Now, investigators in Germany have used gene therapy to
cure 2 of these patients, and for about 2 years, they have
been able to successfully fight off microbial diseases.
o Others Include: Parkinson’s Disease, Hemophilia, and
more…