1. GENE THERAPY

2. • Definition of gene therapy:
“ Novel approach to treat,cure or ultimately
prevent a disease by changing the
expression of a person’s genes”
Dr. Padmesh,V,Slideshare.net

3. Alright,tell me
how you want to
change…!
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4. Gene Therapy
• Tell me how do you want to change
– Replacing a mutated gene that causes
disease with a healthy copy of the gene.
– Inactivating, or “knocking out,” a mutated
gene that is functioning improperly.
– Introducing a new gene into the body to help
fight a disease.
Dr. Padmesh V, Slideshare.net

5. STEPS IN GENE THERAPY:
2. Identification of the defective gene.
3. Cloning of normal healthy gene.
4. Identification of target cell / tissue / organ.
5. Insertion of the normal functional gene into the host
DNA.
METHOD:
Introduction of FUNCTIONAL GENES into appropriate cells
Transferred gene (TRANSGENE) encodes & produces proteins
The Proteins encoded by Transgene corrects the disorder
Dr. Padmesh V, Slideshare.net

6. Cloned gene
(normal allele) 1 An RNA version of
a normal human
gene is inserted
into a retrovirus.
RNA genome of virus
Retrovirus
2 Bone marrow cells
are infected with
the virus.
3 Viral DNA carrying the
human gene inserts into
the cell’s chromosome.
Bone marrow
cell from the patient
4 The engineered
cells are injected
into the patient.
Bone
marrow

7. Cloned gene
(normal allele) 1 An RNA version of
a normal human
gene is inserted
into a retrovirus.
RNA genome of virus
Retrovirus

8. 2 Bone marrow cells
are infected with
the virus.
3 Viral DNA carrying the
human gene inserts into
the cell’s chromosome.
Bone marrow
cell from the patient
4 The engineered
cells are injected
into the patient. Bone
marrow

9. ore
e no m !
u ar nant!!
Yo m i
Do
Google images