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What is Gene Therapy

• It is a technique for correcting defectivegenes that are responsible for diseasedevelopment

• There are four approaches:1. A normal gene inserted to compensate for

a nonfunctional gene.

2. An abnormal gene traded for a normal gene

. An abnormal gene repaired throughselective reverse mutation

!. "hange the regulation of gene pairs

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 The #eginning....

• In the 1$%&s' (cientists began to loo)into gene therapy. – They *ould insert human genes into a

bacteria cell.

 – Then the bacteria cell *ould transcribeand translate the information into a

protein – Then they *ould introduce the protein

into human cells

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+o* It Wor)s

• A vector delivers the therapeutic geneinto a patient,s target cell

•  The target cells become infected *iththe viral vector

•  The vector,s genetic material isinserted into the target cell

• -unctional proteins are created fromthe therapeutic gene causing the cellto return to a normal state

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What is antisense therapy

• Antisense drugs/therapy are a ne*generation of gene0silencingtherapeutic agents *ith potential for

targeted causal treatment of as yetincurable diseases. Antisensetherapy is a form of treatment for

genetic disorders or infections.

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+o* it *or)

•  This synthesied nucleic acid istermed an anti0senseoligonucleotide because its base

sequence is complementary to thegene3s messenger 45A 6m45A7'*hich is called the sense sequence

6so that a sense segment of m45A 830AAGG9"03 *ould be bloc)ed bythe anti0sense m45A segment 30

99""AG083 7.

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• Antisense oligonucleotides havebeen researched as potential drugsfor diseases such as cancers 

6including lung cancer' colorectalcarcinoma' pancreatic carcinoma'malignant glioma and malignant

melanoma7' diabetes' Amyotrophiclateral sclerosis 6A(7' Duchennemuscular dystrophy and diseases

such as asthma' arthritis and

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"ategoriantion of antisense

therapy

Antisense classify into three typesof antisense oligonucleotides,ribozymes and anti-gene.

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