GENE THERAPY

WHAT DOES GENE THERAPY MEAN?

• There wasn’t treatment for “hereditary disease”: genetic

disease wasn’t treated.
• To help these patients, came one of the most modern direction
of medical genetics – gene therapy. Gene therapy is a set of
methods of genetic engineering and medicine, directed at
treatment of genetic disease.
• Gene therapy – is a method of treatment for “hereditary
disease” by gene.
 HOW DOES IT WORK?

• In some cells of a patient man add that gene, which is affected.

They use “carrier viruses” to embed cell into the gene. The
idea of a gene therapy was arose after that when was opened
viruses, which are causes the cancer: they have got an ability
to integrate into the gene and stay there throughout the life of
the cell.
 DISEASES THAT CAN BE TREATED BY
GENE THERAPY

Sickle cell anemia(blood disease)

Alzheimer disease(when you lose your memory, because your

brain stop performing his functions)
 DISEASES

Lesh - nihan syndrome(mental deficiency)

• Parkinson disease(motor functions are impaired in brain)

 DISEASES

• Emphysema(the lungs are inflated, the person has problems

with breath and always coughs)

Thalassemia(because of this disease you have dizziness,

enlargement of spleen)
 DISEASES

• Gurler syndrome(unusual , big head , big forehead , short

neck , short height)

Hunter’s disease(thickening of the lips , tongue and nose)

 ASHANTI DE SILVA

•Ashanti de Silva—the young woman who could treated by gene therapy.

Her health was very bad. When she was 2 y.o , doctors diagnosed
SCI(severe combined immunodeficiency). The reason of this was inborn
disease was“a breakdown in the gene" , which plays a big role in the cell
of immunity. Without treatment most children died, because they were
very weak.
•The doctor Anderson wants to make an experiment and treats Ashanti with
gene therapy. Ashanti’s parents agreed with Anderson. Anderson took from
her blood defective cells, multiplied them in a test tube and entered them
back to the blood. After a year the number of healthy cells were more than
diseased cells. She became healthy and started living a normal life.