1. GENE THERAPY FOR ADENOSINE DEAMINASE DEFICIENCY
Presented by
SAMINA PARVEEN
ROLL NO. 216
UID:

2. GENE THERAPY
It is the curing of a genetic disorder by introducing into the individual a normal gene in order to replace or overcome the effects of a mutant gene.
There are two types of gene therapy:
Somatic cell therapy
Germ line cell therapy

3. TYPES OF GENE THERAPY Somatic cell therapy Germ line cell therapy
Somatic cells are modified genetically to prevent a genetic defect in the offspring.
Germ line cells are used to correct a genetic defect.

4. ADENOSINE DEAMINASE DEFICIENCY{ADA}
ADA deficiency is one form of SCID (severe combined immunodeficiency), a type of disorder that affects the immune system.
The disease is caused by a mutation in a gene on chromosome 20, which codes for the enzyme adenosine deaminase (ADA). Without this enzyme, the body is unable to break down a toxic substance called deoxyadenosine. The toxin builds up and destroys infection-fighting immune cells called T and B lymphocytes

6. GENE THERAPY FOR ADA TREATMENT
First clinical gene therapy was given in 1990 to a 4 year old suffering from ADA.
Steps for carrying it out-
T cells were isolated from the girl and grown
The normal ADA gene was introduced using a viral vector
The “engineered cells” were then re-introduced into the patient

8. CHALLENGES IN GENE THERAPY
Gene delivery and activation
Immune Response
Case of Jesse Gelsinger
Disrupting important cells in target tissue

9. REFERANCES BOOK i-Genetics by Peter Russell Internet sites