A DREAM COMES TRUE

gene therapy

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Points of Discussion:
o o o o o o

History of gene therapy gene therapy working of gene therapy Types of vectors Gene therapy & diseases Advantages/disadvantages of gene therapy

History of gene therapy

1990 the first gene therapy clinical trial began.

1999 gene therapy suffered a major setback with the death of 18-year-old Jesse Gelsinger January2003 Another major blow came April 2003 the FDA eased the ban on gene therapy trials using retroviral vectors in blood stem cells.

Gene therapy definition

Gene therapy
is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery.

Researchers are testing several approaches to gene therapy, including: 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

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How does the gene therapy working?

A normal gene is inserted into the genome to replace an "abnormal, disease-causing gene. A carrier molecule called a vector must be used to deliver the therapeutic gene to the patient's target cells. Currently, the most common vector is a virus that has been genetically altered to carry normal human DNA.

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T y p e s o f v e c t o r s

NA Vr n c od i e a ne r t k ar e i n vd l o i v n r D vi ac er N l A cu t s oe r s s

Retroviruses - A class of viruses that can create doublestranded DNA copies of their RNA genomes. These copies of its genome can be integrated into the chromosomes of host cells. Human immunodeficiency virus (HIV) is a retrovirus. Adenoviruses - A class of viruses with double-stranded DNA genomes that cause respiratory, intestinal, and eye infections in humans. The virus that causes the common cold is an adenovirus.

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Gene therapy & diseases

First gene therapy is made to (OTCD) OrnithineTranscarboxylase Deficiency

gene therapy for (AIDS)

gene therapy for (CANCER)

Cancer
is a class of diseases in which a group of cells display uncontrolled growth (division beyond the normal limits), invasion (intrusion on and destruction of adjacent tissues), and sometimes metastasis (spread to other locations in the body via lymph or blood). These three malignant properties of cancers differentiate them from benign tumors , which are self-limited, and do not invade or metastasize. Most cancers form a tumor but some, like leukemia, do not.

Cancers are caused by a series of mutations. Each mutation alters the behavior of the cell somewhat.

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How is gene therapy being studied in the treatment of cancer?

gene therapy techniques under study for treating cancer

replacing missing or altered genes with healthy genes. improve a patient's immune response to cancer. insertion of genes into cancer cells . introduce suicide genes into a patient's cancer cells. use of gene therapy to prevent cancer cells from developing new blood vessels (angiogenesis ).

Advantages/disadvantages of gene therapy

Advantages

effective stable and long-term expression of the protein (de Almeida et al., 2001) localized to the injection site continuous supply of the protein of choice (as opposed to changing concentration gradients involved with bolus injections (Hermens & Verhaagen, 1998)) avoidance of tissue compression, inflammation and edema often associated with protein infusion devices (Jones & Tuszynski, 2001).

Disadvantage s
Pathogenicity (Verma & Somia, 1997) immune response (Hendriks et al., 2004) Incorporation into the host genome may lead to derepression of tumour suppression genes (Tuszynski, 2005) repression of tumor suppression genes (Tuszynski, 2005) Difficult to control the exact amount of viral product and the duration of expression (Aebischer & Ridet, 2001

QUESTIONS

Under Supervision of dr. Ebtissam Hussein A. Hussein Presented by

Hebashy Mokhtar

Tarek Mohammed Khaled Sameer

References
http://ghr.nlm.nih.gov/handbook/therapy/genetherapy

http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherap

http://www.cancer.gov/cancertopics/factsheet/Therapy/gene

http://www.abcam.com/index.html?pageconfig=resource&rid=10201&pid=7 www.Dnatube.com www.wikipidia.com

Prepared by khaled
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