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Presented by:

Nurul Miza Shasheiha binti Abdul Mutalib -5089Wan Fathiah Nasuha binti Wan Nudri -5117Nurul Husna binti Muryadi -5088-

Nur Amalin Shafirah binti
Nur Farzana Sahirah binti Ariffuddin

• Definiton: an experimental technique for correcting defective genes that are responsible for disease development • The most common form of gene therapy involves inserting a normal gene to replace an abnormal gene • Other approaches used:  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.

• Researchers are studying gene therapy for a number of diseases, such as  Severe combined immuno-deficiencies (SCID)

 Hemophilia
 Parkinson's disease  Cancer  HIV

• 1960: The concepts of Gene Therapy was introduced

• 1970: Friedmann and Roblin author of a paper in Science titled "Gene therapy for human genetic disease?” cite the first attempt to perform gene therapy
• 1990:

 The first approved gene therapy case at the National Institute of Health, U.K. It was performed on a four year old girl named Ashanti DaSilva. It was a treatment for a genetic defect that left her with an immune system deficiency  New gene therapy approach repairs errors in messenger RNA derived from defective genes. This technique has the potential to treat the blood disorder Thalassaemia, Cystic fibrosis, and some cancers
 Sickle cell disease is successfully treated in mice

This study constitutes one of the first demonstrations that gene therapy can be effective in treating cancer. Italy performed the first procedure of gene therapy using hematopoietic stem cells as vectors to deliver genes intended to correct hereditary diseases • 1999: Death of Jesse Gelsinger in a gene-therapy experiment resulted in a significant setback to gene therapy research in the United States • 2006: Scientists at the National Institutes of Health (Bethesda. Milan.2011: Research is still ongoing and the number of diseases that has been treated successfully by gene therapy increases. Maryland) have successfully treated metastatic melanoma in two patients. Gero Hutter has cured a man from HIV using gene therapy .• 1992: Doctor Claudio Bordignon working at the Vita-Salute San Raffaele University. • 2007. Retinal disease Colour blindness Adrenoleukodystrophy • 2011: Medical community accepted that it can cure HIV as in 2008.


Also raises controversy: Some people view this type of therapy as unnatural. Potential for offering a permanent therapeutic effect for all who inherit the target gene. Others have concerns about the technical aspects.      Result in permanent changes. Possibility of eliminating some diseases from a particular family. . and liken it to "playing God”.


and certain infectious diseases. including cystic fibrosis. cancer.  Appropriate and acceptable for many disorders.  Short-lived because the cells of most tissues ultimately die and are replaced by new cells.  . and is not passed to future generations.  Transporting the gene to the target cells or tissue is also problematic.Affects only the targeted cells in the patient. muscular dystrophy.





The two major classes of methods :  recombinant viruses – VIRAL VECTOR  naked DNA or DNA complexes – NONVIRAL VECTOR  . which can be accomplished by a number of methods. GT utilizes the delivery of DNA into cells.

VIRAL VECTOR .Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner. Scientists have tried to harness this ability by manipulating the viral genome to remove diseasecausing genes and insert therapeutic ones .

  The viruses used are altered to make them safe. Virus bind to their hosts and introduce their genetic material into the host cell. Plausible strategy for gene therapy. although some risks still exist with gene therapy. by removing the viral DNA and using the virus as a vehicle to deliver the therapeutic DNA. VIRUS .

Other viruses used as vectors include adeno-associated viruses. pox viruses. Many GT clinical trials rely on retroviruses or adenoviruses to deliver the desired gene. lentiviruses. and whether they alter the cell’s DNA permanently or temporarily   TYPES OF VIRUS . and herpes viruses. Differ in how well they transfer genes to the cells they recognize and are able to infect. alphaviruses.



 Are a tool commonly used by molecular biologists to deliver genetic material into cells. Viruses have evolved specialized molecular mechanisms to efficiently transport their genomes inside the cells they infect.    VIRAL VECTOR . Can be performed in vivo or in vitro. Delivery of genes by a virus is termed transduction and the infected cells are described as transduced.


 Methods of non-viral gene delivery have also been explored using physical (carrier-free gene delivery) and chemical approaches (synthetic vector-based gene delivery). NON VIRAL VECTOR .

Physical approaches. including  Needle injection  Electroporation  Gene gun  Ultrasound  Hydrodynamic delivery employ a physical force that permeates the cell membrane and facilitates intracellular gene transfer  PHYSICAL METHOD .

however. I. NAKED DNA . The simplest method of non-viral transfection. Clinical trials carried out of intramuscular injection of a naked DNA plasmid have occurred with some success. the expression has been very low in comparison to other methods of transfection.

. does not compare to that of the other methods. which shoots DNA coated gold particles into the cell using high pressure gas. This success. leading to research into more efficient methods for delivery of the naked DNA such as electroporation and the use of a "gene gun". however.


      DNA must be protected from damage & its entry into the cell must be facilitated Plasmid DNA can be covered with lipids in an organized structure like a micelle or a liposome complexed with DNA it is called a lipoplex 3 types of lipids: anionic (negatively charged) neutral cationic (positively charged) LIPOPLEXES .

there is little toxicity associated with them. -The cationic lipids also protect against degradation of the DNA by the cell. -naturally complex with the negatively charged DNA. -they are compatible with body fluids -there was a possibility of adapting them to be tissue specific -they are complicated turned to the cationic versions.      Initially. anionic and neutral lipids : -were used for the construction of lipoplexes for synthetic vectors. -but. -their charge they interact with the cell membrane -endocytosis of the lipoplex occurs -DNA is released into the cytoplasm.       . Cationic lipids. due to their positive charge.

 useful in transfecting respiratory epithelial cells.  .Common used of lipoplexes In gene transfer into cancer cells. so they may be used for treatment of genetic respiratory diseases such as cystic fibrosis. where the supplied genes have activated tumor suppressor control genes in the cell  decrease the activity of oncogenes.

the process by which the polyplex enters the cell)  POLYPLEXES .Complexes of polymers with DNA are called polyplexes  consist of cationic polymers and their production is regulated by ionic interactions.  large difference compared to lipoplexes is that polyplexes cannot release their DNA load into the cytoplasm.  End= co-transfection with endosome-lytic agents such as inactivated adenovirus must occur (to lyse the endosome that is made during endocytosis.


GENE THERAPY CURES BLINDNESS  Cure blindness of inherited condition  Leber’s conginetal amaurosis .The condition appears at birth or in the first few months of life and causes progressive worse and loss of vision.inherited disease caused by an abnormality in a gene called RPE65. . .

HOW IT WORKS??  used harmless viruses  enable access to the cells beneath the retinas of patients  By using a very fine needle -safe in an extremely fragile tissue and can improve vision in a condition previously considered wholly . •


and rigidity  Main. .overactive brain region: the subthalamic nucleus should be introduced with gene  that would produce GABA—an inhibitory chemical—then they could potentially quiet that brain region and alleviate tremors. motor skill problems.GENE THERAPY REDUCES PARKINSON’S DISEASE SYMPTOMS  it significantly improved the weakness of the symptoms such as tremors.

used a harmless. inactive virus [AAV-2 GAD]  Deliver the GAD gene into patient’s subthalamic nucleus  The gene instructs cells to begin making GABA neurotransmitters to re-establish the normal chemical balance that becomes dysfunctional as the disease progresses .HOW IT WORKS??  Done with local anesthesia.

ADVANTAGES OF GENE THERAPY • Give a chance of a normal life to baby born with genetic disease. it could save many lives . • For certain disease that do not have any cure except gene therapy. • Give hope of healthy life to cancer patient.

• Cosmetic industry may monopolized this gene therapy if it is used in enhancing beauty and in vanishing the aging effect. rather than used for treatment of a disease. • The cost is very high and the patient might need an insurance to cover the treatment. . • May increase rate of abortion if prenatal test regarding baby with genetic disease is done. screening and research in finding the availability of certain gene is very controversy.DISADVANTAGES OF GENE THERAPY • The genetic testing.

ETHICAL QUESTIONS SURROUNDING GENE THERAPY • How can “good” and “bad” uses of gene therapy be distinguished? • Who decides which traits are normal and which constitute a disability or disorder? • Will the therapy only benefit the wealthy due to its high cost? • Could the widespread use of gene therapy make the society less accepting of people who are different? • Should people be allowed to use gene therapy to enhance basic human traits such as height. or athletic ability? . intelligence.