What is Gene Therapy

It is a technique for correcting defective

genes that are responsible for disease
development
There are four approaches:
1. A normal gene inserted to compensate for
a nonfunctional gene.
2. An abnormal gene traded for a normal gene
3. An abnormal gene repaired through
selective reverse mutation
4. Change the regulation of gene pairs

The Beginning....
In the 1980s, Scientists began to look
into gene therapy.
They would insert human genes into a
bacteria cell.
Then the bacteria cell would transcribe
and translate the information into a
protein
Then they would introduce the protein
into human cells

How It Works
A vector delivers the therapeutic gene
into a patients target cell
The target cells become infected with
the viral vector
The vectors genetic material is
inserted into the target cell
Functional proteins are created from
the therapeutic gene causing the cell
to return to a normal state

What is antisense therapy?

Antisense drugs/therapy are a new

generation of gene-silencing
therapeutic agents with potential for
targeted causal treatment of as yet
incurable diseases. Antisense
therapy is a form of treatment for
genetic disorders or infections.

How it work?
This synthesized nucleic acid is
termed an "anti-sense"
oligonucleotide because its base
sequence is complementary to the
gene's messenger RNA (mRNA),
which is called the "sense" sequence
(so that a sense segment of mRNA "
5'-AAGGUC-3' " would be blocked by
the anti-sense mRNA segment " 3'UUCCAG-5' ").

 Antisense oligonucleotides have

been researched as potential drugs
for diseases such as cancers
(including lung cancer, colorectal
carcinoma, pancreatic carcinoma,
malignant glioma and malignant
melanoma), diabetes, Amyotrophic
lateral sclerosis (ALS), Duchenne
muscular dystrophy and diseases
such as asthma, arthritis and

Categorizantion of antisense
therapy
Antisense classify into three 3 types
of antisense oligonucleotides,
ribozymes and anti-gene.