GENE THERAPY: TREATING

YOUR GENES
 - IS THE TRANSFER
WHAT IS GENE
THERAPY?
OF GENETIC
MATERIAL (DNA)
FOR THE
PURPOSE OF
TREATING HUMAN
DISEASES.
DOMINANT GENE -
traits are those that are
expressed or seen.

RECESSIVE GENE
a gene that can be
masked by a
dominant gene.

https://www.education.com/science-fair/article/biology_it-
takes/
 Genetic diseases may be classified into 4 categories:

1. SINGLE-GENE CHANGES SINGLE LOCUS (GENE)

IS DEFECTIVE AND RESPONSIBLE FOR THE
DISEASES; IT IS ALSO 100% HERITABLE.

EXAMPLE:

https://www.bluebirdbio.com/patients-families/sickle-cell-disease/
 Sickle cell anemia is a disease in which your body
produces abnormally shaped red blood cells. The
cells are shaped like a crescent or sickle. They
don't last as long as normal, round red blood
cells. This leads to anemia. The sickle cells also
get stuck in blood vessels, blocking blood flow.
This can cause pain and organ damage.

Sickle cell anemia has no widely available cure.

Treatments can help relieve symptoms and lessen
complications.
https://www.slideshare.net/MohamedElasalyPTCKTP/sickle-cell-anemia-75934817
2. MULTIGENE DISORDER TWO OR MORE
GENES ARE DEFECTIVE AND RESPONSIBLE FOR
THE DISEASE; LESS THAN 100% HERITABLE.
THIS DISEASE MAY BE DEPENDENT ON
ENVIRONMENTAL FACTORS AND LIFESTYLE.

EXAMPLE:

http://www.medicinenet.com/heart_attack/article.htm
3. MITOCHONDRIAL DISORDERS MUTATIONS IN THE
MITOCHONDRIAL DNA CAUSE DISEASES THAT AFFECT
MANY ORGAN SYSTEM.
The mitochondria, they are known as the "powerhouse of
the cell."
In the cells, throughout our bodies are responsible for
creating 90% of the energy needed to sustain life and
support organ function. When mitochondria malfunction,
organs start to fail people get sick, and even die.
SYMPTOMS:

http://mitochondrialdiseases.org/mitochondrial-disease/
4. CHROMOSOME ABNORMALITIES SOMETIMES
COMPLETE CHROMOSOMES OR PART OF THE
CHROMOSOME ARE MISSING, DUPLICATED OR
MODIFIED IN SOME WAY.

EXAMPLE:

https://www.slideshare.net/twgriffin10/down-syndrome-4993709
SOMATIC CELL GENE THERAPY:

https://www.slideshare.net/syeimy/gene-therapy-ppt
Three routes for somatic gene therapy:

EX VIVO IN SITU IN VIVO

PLACED DIRECTLY INTO

THE AFFECTED TISSUES

https://www.slideshare.net/iftheqharahmad/gene-therapy-42079030
Are you in favor
to use gene
therapy to treat a
disease? Explain
your answer.